Informacja

Drogi użytkowniku, aplikacja do prawidłowego działania wymaga obsługi JavaScript. Proszę włącz obsługę JavaScript w Twojej przeglądarce.

Przeglądasz jako GOŚĆ

Wyszukujesz frazę ""controlled study"" wg kryterium: Temat


Tytuł :
Reliable, cheap, fast and few: What is the best study for assessing medical practices? Randomized controlled trials or synthetic control arms?
Autorzy :
Prasad V; Department of Epidemiology and Biostatistics, University of California San Francisco, San Francisco, CA, USA.; Department of Medicine, University of California San Francisco, San Francisco, CA, USA.
Pokaż więcej
Źródło :
European journal of clinical investigation [Eur J Clin Invest] 2021 Aug; Vol. 51 (8), pp. e13580. Date of Electronic Publication: 2021 Jun 19.
Typ publikacji :
Journal Article
MeSH Terms :
Historically Controlled Study*/economics
Observational Studies as Topic*/economics
Randomized Controlled Trials as Topic*/economics
Humans
Czasopismo naukowe
Tytuł :
Improved management of placenta accreta spectrum disorders: experience from a single institution.
Autorzy :
Sylvester-Armstrong K; University of Florida College of Medicine, Gainesville, FL, USA.
Reeder C; University of Florida College of Medicine, Gainesville, FL, USA.
Patrick K; University of Florida College of Medicine, Gainesville, FL, USA.
Genc MR; University of Florida College of Medicine, Gainesville, FL, USA.
Pokaż więcej
Źródło :
Journal of perinatal medicine [J Perinat Med] 2021 Dec 16; Vol. 50 (3), pp. 286-293. Date of Electronic Publication: 2021 Dec 16 (Print Publication: 2022).
Typ publikacji :
Journal Article
MeSH Terms :
Clinical Protocols*
Placenta Accreta/*surgery
Adult ; Blood Loss, Surgical ; Blood Transfusion/statistics & numerical data ; Cesarean Section ; Female ; Historically Controlled Study ; Humans ; Postpartum Hemorrhage/prevention & control ; Pregnancy
Czasopismo naukowe
Tytuł :
Evaluating the Effect of Pravastatin in Early-Onset Fetal Growth Restriction: A Nonrandomized and Historically Controlled Pilot Study.
Autorzy :
Mendoza M; Department of Obstetrics, Maternal Fetal Medicine Unit, Hospital Universitari Vall d'Hebron, Universitat Autònoma de Barcelona, Barcelona, Spain.
Ferrer-Oliveras R; Department of Obstetrics, Maternal Fetal Medicine Unit, Hospital Universitari Vall d'Hebron, Universitat Autònoma de Barcelona, Barcelona, Spain.
Bonacina E; Department of Obstetrics, Maternal Fetal Medicine Unit, Hospital Universitari Vall d'Hebron, Universitat Autònoma de Barcelona, Barcelona, Spain.
Garcia-Manau P; Department of Obstetrics, Maternal Fetal Medicine Unit, Hospital Universitari Vall d'Hebron, Universitat Autònoma de Barcelona, Barcelona, Spain.
Rodo C; Department of Obstetrics, Maternal Fetal Medicine Unit, Hospital Universitari Vall d'Hebron, Universitat Autònoma de Barcelona, Barcelona, Spain.
Carreras E; Department of Obstetrics, Maternal Fetal Medicine Unit, Hospital Universitari Vall d'Hebron, Universitat Autònoma de Barcelona, Barcelona, Spain.
Alijotas-Reig J; Department of Medicine, Systemic Autoimmune Diseases Unit, Hospital Universitari Vall d'Hebron, Universitat Autònoma de Barcelona, Barcelona, Spain.
Pokaż więcej
Źródło :
American journal of perinatology [Am J Perinatol] 2021 Dec; Vol. 38 (14), pp. 1472-1479. Date of Electronic Publication: 2020 Jul 02.
Typ publikacji :
Journal Article
MeSH Terms :
Ultrasonography, Prenatal*
Fetal Growth Retardation/*drug therapy
Pravastatin/*therapeutic use
Biomarkers/blood ; Birth Weight ; Female ; Fetal Development/drug effects ; Fetal Growth Retardation/diagnostic imaging ; Historically Controlled Study ; Humans ; Infant, Newborn ; Pilot Projects ; Placenta Growth Factor/blood ; Pregnancy ; Pregnancy Outcome ; Ultrasonography, Doppler ; Vascular Endothelial Growth Factor Receptor-1/blood
Czasopismo naukowe
Tytuł :
Rationale and design of individualized quality improvement based on the Computer Analysing system to improve Stroke management quality Evaluation (CASE): a multicenter historically controlled study.
Autorzy :
Chen Y; Department of Neurology, The Second Affiliated Hospital of Zhejiang University School of Medicine, 88# Jiefang Road, Hangzhou, 310009, China.
Zhong W; Department of Neurology, The Second Affiliated Hospital of Zhejiang University School of Medicine, 88# Jiefang Road, Hangzhou, 310009, China.
Gong X; Department of Neurology, The Second Affiliated Hospital of Zhejiang University School of Medicine, 88# Jiefang Road, Hangzhou, 310009, China.
Hu H; Department of Neurology, The Second Affiliated Hospital of Zhejiang University School of Medicine, 88# Jiefang Road, Hangzhou, 310009, China.
Yan S; Department of Neurology, The Second Affiliated Hospital of Zhejiang University School of Medicine, 88# Jiefang Road, Hangzhou, 310009, China.
Zhang X; Department of Neurology, The Second Affiliated Hospital of Zhejiang University School of Medicine, 88# Jiefang Road, Hangzhou, 310009, China.
Chen Z; Department of Neurology, The Second Affiliated Hospital of Zhejiang University School of Medicine, 88# Jiefang Road, Hangzhou, 310009, China.
Zhou Y; Department of Neurology, The Second Affiliated Hospital of Zhejiang University School of Medicine, 88# Jiefang Road, Hangzhou, 310009, China.
Lou M; Department of Neurology, The Second Affiliated Hospital of Zhejiang University School of Medicine, 88# Jiefang Road, Hangzhou, 310009, China. .
Pokaż więcej
Źródło :
Trials [Trials] 2020 Jul 24; Vol. 21 (1), pp. 677. Date of Electronic Publication: 2020 Jul 24.
Typ publikacji :
Journal Article
MeSH Terms :
Brain Ischemia*/diagnosis
Brain Ischemia*/therapy
Historically Controlled Study*
Quality of Health Care*
Stroke*/diagnosis
Stroke*/therapy
China ; Humans ; Multicenter Studies as Topic ; Prospective Studies ; Quality Improvement
Czasopismo naukowe
Tytuł :
Group sequential design for historical control trials using error spending functions.
Autorzy :
Wu J; Biostatistics and Bioinformatics Shared Resource Facility, Markey Cancer Center, University of Kentucky, Lexington, KY, USA.
Li Y; Department of Biostatistics, St. Jude Children's Research Hospital, Memphis, TN, USA.
Pokaż więcej
Źródło :
Journal of biopharmaceutical statistics [J Biopharm Stat] 2020 Mar; Vol. 30 (2), pp. 351-363. Date of Electronic Publication: 2019 Nov 12.
Typ publikacji :
Journal Article; Research Support, N.I.H., Extramural
MeSH Terms :
Computer Simulation/*statistics & numerical data
Historically Controlled Study/*statistics & numerical data
Research Design/*statistics & numerical data
Data Interpretation, Statistical ; Historically Controlled Study/methods ; Humans
Czasopismo naukowe
Tytuł :
Management of asymptomatic hypoglycemia with 40% oral dextrose gel in near term at-risk infants to reduce intensive care need and promote breastfeeding.
Autorzy :
Meneghin F; NICU, Department of Pediatrics, V. Buzzi Children's Hospital, ASST-FBF-Sacco, Milan, Italy. .
Manzalini M; Department of Pediatrics, University of Milan, V. Buzzi Children's Hospital, ASST-FBF-Sacco, Milan, Italy.
Acunzo M; Department of Pediatrics, University of Milan, V. Buzzi Children's Hospital, ASST-FBF-Sacco, Milan, Italy.
Daniele I; NICU, Department of Pediatrics, V. Buzzi Children's Hospital, ASST-FBF-Sacco, Milan, Italy.
Bastrenta P; NICU, Department of Pediatrics, V. Buzzi Children's Hospital, ASST-FBF-Sacco, Milan, Italy.
Castoldi F; NICU, Department of Pediatrics, V. Buzzi Children's Hospital, ASST-FBF-Sacco, Milan, Italy.
Cavigioli F; NICU, Department of Pediatrics, V. Buzzi Children's Hospital, ASST-FBF-Sacco, Milan, Italy.
Zuccotti GV; Department of Pediatrics, University of Milan, V. Buzzi Children's Hospital, ASST-FBF-Sacco, Milan, Italy.
Lista G; NICU, Department of Pediatrics, V. Buzzi Children's Hospital, ASST-FBF-Sacco, Milan, Italy.
Pokaż więcej
Źródło :
Italian journal of pediatrics [Ital J Pediatr] 2021 Oct 09; Vol. 47 (1), pp. 201. Date of Electronic Publication: 2021 Oct 09.
Typ publikacji :
Journal Article
MeSH Terms :
Glucose/*administration & dosage
Hypoglycemia/*therapy
Sweetening Agents/*administration & dosage
Administration, Oral ; Asymptomatic Diseases ; Breast Feeding/statistics & numerical data ; Female ; Gels ; Historically Controlled Study ; Humans ; Infant, Newborn ; Infusions, Intravenous ; Intensive Care Units, Neonatal ; Male ; Patient Admission/statistics & numerical data ; Prospective Studies
Czasopismo naukowe
Tytuł :
Placenta Accreta Spectrum Treatment With Intraoperative Multivessel Embolization: the PASTIME protocol.
Autorzy :
Melber DJ; Division of Maternal-Fetal Medicine, Department of Obstetrics, Gynecology and Reproductive Sciences, University of California San Diego, San Diego, CA. Electronic address: .
Berman ZT; Division of Interventional Radiology, Department of Radiology, University of California San Diego, San Diego, CA.
Jacobs MB; Division of Maternal-Fetal Medicine, Department of Obstetrics, Gynecology and Reproductive Sciences, University of California San Diego, San Diego, CA.
Picel AC; Division of Interventional Radiology, Department of Radiology, Stanford University, Stanford, CA.
Conturie CL; Division of Maternal-Fetal Medicine and Obstetrics, Department of Obstetrics and Gynecology, Stanford University, Stanford, CA.
Zhang-Rutledge K; Division of Maternal-Fetal Medicine, Department of Obstetrics, Gynecology and Reproductive Sciences, University of California San Diego, San Diego, CA.
Binder PS; Division of Gynecologic Oncology, Department of Obstetrics, Gynecology and Reproductive Sciences, University of California San Diego, San Diego, CA.
Eskander RN; Division of Gynecologic Oncology, Department of Obstetrics, Gynecology and Reproductive Sciences, University of California San Diego, San Diego, CA.
Roberts AC; Division of Interventional Radiology, Department of Radiology, University of California San Diego, San Diego, CA.
McHale MT; Division of Gynecologic Oncology, Department of Obstetrics, Gynecology and Reproductive Sciences, University of California San Diego, San Diego, CA.
Ramos GA; Division of Maternal-Fetal Medicine, Department of Obstetrics, Gynecology and Reproductive Sciences, University of California San Diego, San Diego, CA.
Ballas J; Division of Maternal-Fetal Medicine, Department of Obstetrics, Gynecology and Reproductive Sciences, University of California San Diego, San Diego, CA.
Kelly TF; Division of Maternal-Fetal Medicine, Department of Obstetrics, Gynecology and Reproductive Sciences, University of California San Diego, San Diego, CA.
Pokaż więcej
Źródło :
American journal of obstetrics and gynecology [Am J Obstet Gynecol] 2021 Oct; Vol. 225 (4), pp. 442.e1-442.e10. Date of Electronic Publication: 2021 Jul 07.
Typ publikacji :
Journal Article
MeSH Terms :
Iliac Artery*
Cesarean Section/*methods
Erythrocyte Transfusion/*statistics & numerical data
Hysterectomy/*methods
Intraoperative Care/*methods
Placenta Accreta/*therapy
Uterine Artery Embolization/*methods
Uterine Hemorrhage/*prevention & control
Adult ; Apgar Score ; Balloon Occlusion ; Blood Loss, Surgical/statistics & numerical data ; Combined Modality Therapy ; Embolization, Therapeutic/methods ; Female ; Gestational Age ; Historically Controlled Study ; Humans ; Intensive Care Units, Neonatal ; Length of Stay/statistics & numerical data ; Operative Time ; Pregnancy ; Radiography, Interventional ; Shock, Hemorrhagic/epidemiology ; Shock, Hemorrhagic/mortality ; Uterine Hemorrhage/therapy
Czasopismo naukowe
Tytuł :
Risdiplam-Treated Infants with Type 1 Spinal Muscular Atrophy versus Historical Controls.
Autorzy :
Darras BT; From the Department of Neurology, Boston Children's Hospital, Harvard Medical School, Boston (B.T.D.); the Developmental Neurology Unit, Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan (R.M., G.B.), and the Center of Translational and Experimental Myology, IRCCS Istituto Giannina Gaslini, Genoa (C.B.) - both in Italy; the Department of Developmental Neurology, Medical University of Gdańsk, Gdańsk, Poland (M.M.-B.); the Paediatric Gait Analysis Service of New South Wales, the Children's Hospital at Westmead and the University of Sydney, Sydney (K.R.); the Department of Pediatrics, Peking University First Hospital, Beijing (H.X.), and Children's Hospital of Fudan University, Shanghai (Y.W.) - both in China; the Department of Neurology, Faculdade de Medicina, Universidade de São Paulo, São Paulo (E.Z.); the Dubowitz Neuromuscular Centre, National Institute for Health Research Great Ormond Street Hospital Biomedical Research Centre, University College London Great Ormond Street Institute of Child Health, and Great Ormond Street Hospital for Children NHS Foundation Trust, London (G.B.), Roche Products, Welwyn Garden City (M.E.-K.), and the Muscular Dystrophy UK Oxford Neuromuscular Centre, the Department of Paediatrics, University of Oxford, Oxford (L.S.) - all in the United Kingdom; Russian Children Neuromuscular Center, Veltischev Clinical Pediatric Research Institute, Pirogov Russian National Research Medical University, Moscow (D.V.); Pharma Development, Safety (M.G.), Product Development Medical Affairs - Neuroscience and Rare Disease (K.G., P.F.), and Pharma Development Neurology (R.S.S.), F. Hoffmann-La Roche, and Roche Pharmaceutical Research and Early Development, Roche Innovation Center Basel (O.K., H.K.) - both in Basel, Switzerland; the Division of Child Neurology, Centre de Références des Maladies Neuromusculaires, the Department of Pediatrics, University Hospital Liege, University of Liege, Liege, Belgium (L.S.); and I-Motion, Institut de Myologie, Assistance Publique Hôpitaux de Paris, Hôpital Armand Trousseau, Paris (L.S.).
Masson R; From the Department of Neurology, Boston Children's Hospital, Harvard Medical School, Boston (B.T.D.); the Developmental Neurology Unit, Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan (R.M., G.B.), and the Center of Translational and Experimental Myology, IRCCS Istituto Giannina Gaslini, Genoa (C.B.) - both in Italy; the Department of Developmental Neurology, Medical University of Gdańsk, Gdańsk, Poland (M.M.-B.); the Paediatric Gait Analysis Service of New South Wales, the Children's Hospital at Westmead and the University of Sydney, Sydney (K.R.); the Department of Pediatrics, Peking University First Hospital, Beijing (H.X.), and Children's Hospital of Fudan University, Shanghai (Y.W.) - both in China; the Department of Neurology, Faculdade de Medicina, Universidade de São Paulo, São Paulo (E.Z.); the Dubowitz Neuromuscular Centre, National Institute for Health Research Great Ormond Street Hospital Biomedical Research Centre, University College London Great Ormond Street Institute of Child Health, and Great Ormond Street Hospital for Children NHS Foundation Trust, London (G.B.), Roche Products, Welwyn Garden City (M.E.-K.), and the Muscular Dystrophy UK Oxford Neuromuscular Centre, the Department of Paediatrics, University of Oxford, Oxford (L.S.) - all in the United Kingdom; Russian Children Neuromuscular Center, Veltischev Clinical Pediatric Research Institute, Pirogov Russian National Research Medical University, Moscow (D.V.); Pharma Development, Safety (M.G.), Product Development Medical Affairs - Neuroscience and Rare Disease (K.G., P.F.), and Pharma Development Neurology (R.S.S.), F. Hoffmann-La Roche, and Roche Pharmaceutical Research and Early Development, Roche Innovation Center Basel (O.K., H.K.) - both in Basel, Switzerland; the Division of Child Neurology, Centre de Références des Maladies Neuromusculaires, the Department of Pediatrics, University Hospital Liege, University of Liege, Liege, Belgium (L.S.); and I-Motion, Institut de Myologie, Assistance Publique Hôpitaux de Paris, Hôpital Armand Trousseau, Paris (L.S.).
Mazurkiewicz-Bełdzińska M; From the Department of Neurology, Boston Children's Hospital, Harvard Medical School, Boston (B.T.D.); the Developmental Neurology Unit, Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan (R.M., G.B.), and the Center of Translational and Experimental Myology, IRCCS Istituto Giannina Gaslini, Genoa (C.B.) - both in Italy; the Department of Developmental Neurology, Medical University of Gdańsk, Gdańsk, Poland (M.M.-B.); the Paediatric Gait Analysis Service of New South Wales, the Children's Hospital at Westmead and the University of Sydney, Sydney (K.R.); the Department of Pediatrics, Peking University First Hospital, Beijing (H.X.), and Children's Hospital of Fudan University, Shanghai (Y.W.) - both in China; the Department of Neurology, Faculdade de Medicina, Universidade de São Paulo, São Paulo (E.Z.); the Dubowitz Neuromuscular Centre, National Institute for Health Research Great Ormond Street Hospital Biomedical Research Centre, University College London Great Ormond Street Institute of Child Health, and Great Ormond Street Hospital for Children NHS Foundation Trust, London (G.B.), Roche Products, Welwyn Garden City (M.E.-K.), and the Muscular Dystrophy UK Oxford Neuromuscular Centre, the Department of Paediatrics, University of Oxford, Oxford (L.S.) - all in the United Kingdom; Russian Children Neuromuscular Center, Veltischev Clinical Pediatric Research Institute, Pirogov Russian National Research Medical University, Moscow (D.V.); Pharma Development, Safety (M.G.), Product Development Medical Affairs - Neuroscience and Rare Disease (K.G., P.F.), and Pharma Development Neurology (R.S.S.), F. Hoffmann-La Roche, and Roche Pharmaceutical Research and Early Development, Roche Innovation Center Basel (O.K., H.K.) - both in Basel, Switzerland; the Division of Child Neurology, Centre de Références des Maladies Neuromusculaires, the Department of Pediatrics, University Hospital Liege, University of Liege, Liege, Belgium (L.S.); and I-Motion, Institut de Myologie, Assistance Publique Hôpitaux de Paris, Hôpital Armand Trousseau, Paris (L.S.).
Rose K; From the Department of Neurology, Boston Children's Hospital, Harvard Medical School, Boston (B.T.D.); the Developmental Neurology Unit, Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan (R.M., G.B.), and the Center of Translational and Experimental Myology, IRCCS Istituto Giannina Gaslini, Genoa (C.B.) - both in Italy; the Department of Developmental Neurology, Medical University of Gdańsk, Gdańsk, Poland (M.M.-B.); the Paediatric Gait Analysis Service of New South Wales, the Children's Hospital at Westmead and the University of Sydney, Sydney (K.R.); the Department of Pediatrics, Peking University First Hospital, Beijing (H.X.), and Children's Hospital of Fudan University, Shanghai (Y.W.) - both in China; the Department of Neurology, Faculdade de Medicina, Universidade de São Paulo, São Paulo (E.Z.); the Dubowitz Neuromuscular Centre, National Institute for Health Research Great Ormond Street Hospital Biomedical Research Centre, University College London Great Ormond Street Institute of Child Health, and Great Ormond Street Hospital for Children NHS Foundation Trust, London (G.B.), Roche Products, Welwyn Garden City (M.E.-K.), and the Muscular Dystrophy UK Oxford Neuromuscular Centre, the Department of Paediatrics, University of Oxford, Oxford (L.S.) - all in the United Kingdom; Russian Children Neuromuscular Center, Veltischev Clinical Pediatric Research Institute, Pirogov Russian National Research Medical University, Moscow (D.V.); Pharma Development, Safety (M.G.), Product Development Medical Affairs - Neuroscience and Rare Disease (K.G., P.F.), and Pharma Development Neurology (R.S.S.), F. Hoffmann-La Roche, and Roche Pharmaceutical Research and Early Development, Roche Innovation Center Basel (O.K., H.K.) - both in Basel, Switzerland; the Division of Child Neurology, Centre de Références des Maladies Neuromusculaires, the Department of Pediatrics, University Hospital Liege, University of Liege, Liege, Belgium (L.S.); and I-Motion, Institut de Myologie, Assistance Publique Hôpitaux de Paris, Hôpital Armand Trousseau, Paris (L.S.).
Xiong H; From the Department of Neurology, Boston Children's Hospital, Harvard Medical School, Boston (B.T.D.); the Developmental Neurology Unit, Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan (R.M., G.B.), and the Center of Translational and Experimental Myology, IRCCS Istituto Giannina Gaslini, Genoa (C.B.) - both in Italy; the Department of Developmental Neurology, Medical University of Gdańsk, Gdańsk, Poland (M.M.-B.); the Paediatric Gait Analysis Service of New South Wales, the Children's Hospital at Westmead and the University of Sydney, Sydney (K.R.); the Department of Pediatrics, Peking University First Hospital, Beijing (H.X.), and Children's Hospital of Fudan University, Shanghai (Y.W.) - both in China; the Department of Neurology, Faculdade de Medicina, Universidade de São Paulo, São Paulo (E.Z.); the Dubowitz Neuromuscular Centre, National Institute for Health Research Great Ormond Street Hospital Biomedical Research Centre, University College London Great Ormond Street Institute of Child Health, and Great Ormond Street Hospital for Children NHS Foundation Trust, London (G.B.), Roche Products, Welwyn Garden City (M.E.-K.), and the Muscular Dystrophy UK Oxford Neuromuscular Centre, the Department of Paediatrics, University of Oxford, Oxford (L.S.) - all in the United Kingdom; Russian Children Neuromuscular Center, Veltischev Clinical Pediatric Research Institute, Pirogov Russian National Research Medical University, Moscow (D.V.); Pharma Development, Safety (M.G.), Product Development Medical Affairs - Neuroscience and Rare Disease (K.G., P.F.), and Pharma Development Neurology (R.S.S.), F. Hoffmann-La Roche, and Roche Pharmaceutical Research and Early Development, Roche Innovation Center Basel (O.K., H.K.) - both in Basel, Switzerland; the Division of Child Neurology, Centre de Références des Maladies Neuromusculaires, the Department of Pediatrics, University Hospital Liege, University of Liege, Liege, Belgium (L.S.); and I-Motion, Institut de Myologie, Assistance Publique Hôpitaux de Paris, Hôpital Armand Trousseau, Paris (L.S.).
Zanoteli E; From the Department of Neurology, Boston Children's Hospital, Harvard Medical School, Boston (B.T.D.); the Developmental Neurology Unit, Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan (R.M., G.B.), and the Center of Translational and Experimental Myology, IRCCS Istituto Giannina Gaslini, Genoa (C.B.) - both in Italy; the Department of Developmental Neurology, Medical University of Gdańsk, Gdańsk, Poland (M.M.-B.); the Paediatric Gait Analysis Service of New South Wales, the Children's Hospital at Westmead and the University of Sydney, Sydney (K.R.); the Department of Pediatrics, Peking University First Hospital, Beijing (H.X.), and Children's Hospital of Fudan University, Shanghai (Y.W.) - both in China; the Department of Neurology, Faculdade de Medicina, Universidade de São Paulo, São Paulo (E.Z.); the Dubowitz Neuromuscular Centre, National Institute for Health Research Great Ormond Street Hospital Biomedical Research Centre, University College London Great Ormond Street Institute of Child Health, and Great Ormond Street Hospital for Children NHS Foundation Trust, London (G.B.), Roche Products, Welwyn Garden City (M.E.-K.), and the Muscular Dystrophy UK Oxford Neuromuscular Centre, the Department of Paediatrics, University of Oxford, Oxford (L.S.) - all in the United Kingdom; Russian Children Neuromuscular Center, Veltischev Clinical Pediatric Research Institute, Pirogov Russian National Research Medical University, Moscow (D.V.); Pharma Development, Safety (M.G.), Product Development Medical Affairs - Neuroscience and Rare Disease (K.G., P.F.), and Pharma Development Neurology (R.S.S.), F. Hoffmann-La Roche, and Roche Pharmaceutical Research and Early Development, Roche Innovation Center Basel (O.K., H.K.) - both in Basel, Switzerland; the Division of Child Neurology, Centre de Références des Maladies Neuromusculaires, the Department of Pediatrics, University Hospital Liege, University of Liege, Liege, Belgium (L.S.); and I-Motion, Institut de Myologie, Assistance Publique Hôpitaux de Paris, Hôpital Armand Trousseau, Paris (L.S.).
Baranello G; From the Department of Neurology, Boston Children's Hospital, Harvard Medical School, Boston (B.T.D.); the Developmental Neurology Unit, Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan (R.M., G.B.), and the Center of Translational and Experimental Myology, IRCCS Istituto Giannina Gaslini, Genoa (C.B.) - both in Italy; the Department of Developmental Neurology, Medical University of Gdańsk, Gdańsk, Poland (M.M.-B.); the Paediatric Gait Analysis Service of New South Wales, the Children's Hospital at Westmead and the University of Sydney, Sydney (K.R.); the Department of Pediatrics, Peking University First Hospital, Beijing (H.X.), and Children's Hospital of Fudan University, Shanghai (Y.W.) - both in China; the Department of Neurology, Faculdade de Medicina, Universidade de São Paulo, São Paulo (E.Z.); the Dubowitz Neuromuscular Centre, National Institute for Health Research Great Ormond Street Hospital Biomedical Research Centre, University College London Great Ormond Street Institute of Child Health, and Great Ormond Street Hospital for Children NHS Foundation Trust, London (G.B.), Roche Products, Welwyn Garden City (M.E.-K.), and the Muscular Dystrophy UK Oxford Neuromuscular Centre, the Department of Paediatrics, University of Oxford, Oxford (L.S.) - all in the United Kingdom; Russian Children Neuromuscular Center, Veltischev Clinical Pediatric Research Institute, Pirogov Russian National Research Medical University, Moscow (D.V.); Pharma Development, Safety (M.G.), Product Development Medical Affairs - Neuroscience and Rare Disease (K.G., P.F.), and Pharma Development Neurology (R.S.S.), F. Hoffmann-La Roche, and Roche Pharmaceutical Research and Early Development, Roche Innovation Center Basel (O.K., H.K.) - both in Basel, Switzerland; the Division of Child Neurology, Centre de Références des Maladies Neuromusculaires, the Department of Pediatrics, University Hospital Liege, University of Liege, Liege, Belgium (L.S.); and I-Motion, Institut de Myologie, Assistance Publique Hôpitaux de Paris, Hôpital Armand Trousseau, Paris (L.S.).
Bruno C; From the Department of Neurology, Boston Children's Hospital, Harvard Medical School, Boston (B.T.D.); the Developmental Neurology Unit, Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan (R.M., G.B.), and the Center of Translational and Experimental Myology, IRCCS Istituto Giannina Gaslini, Genoa (C.B.) - both in Italy; the Department of Developmental Neurology, Medical University of Gdańsk, Gdańsk, Poland (M.M.-B.); the Paediatric Gait Analysis Service of New South Wales, the Children's Hospital at Westmead and the University of Sydney, Sydney (K.R.); the Department of Pediatrics, Peking University First Hospital, Beijing (H.X.), and Children's Hospital of Fudan University, Shanghai (Y.W.) - both in China; the Department of Neurology, Faculdade de Medicina, Universidade de São Paulo, São Paulo (E.Z.); the Dubowitz Neuromuscular Centre, National Institute for Health Research Great Ormond Street Hospital Biomedical Research Centre, University College London Great Ormond Street Institute of Child Health, and Great Ormond Street Hospital for Children NHS Foundation Trust, London (G.B.), Roche Products, Welwyn Garden City (M.E.-K.), and the Muscular Dystrophy UK Oxford Neuromuscular Centre, the Department of Paediatrics, University of Oxford, Oxford (L.S.) - all in the United Kingdom; Russian Children Neuromuscular Center, Veltischev Clinical Pediatric Research Institute, Pirogov Russian National Research Medical University, Moscow (D.V.); Pharma Development, Safety (M.G.), Product Development Medical Affairs - Neuroscience and Rare Disease (K.G., P.F.), and Pharma Development Neurology (R.S.S.), F. Hoffmann-La Roche, and Roche Pharmaceutical Research and Early Development, Roche Innovation Center Basel (O.K., H.K.) - both in Basel, Switzerland; the Division of Child Neurology, Centre de Références des Maladies Neuromusculaires, the Department of Pediatrics, University Hospital Liege, University of Liege, Liege, Belgium (L.S.); and I-Motion, Institut de Myologie, Assistance Publique Hôpitaux de Paris, Hôpital Armand Trousseau, Paris (L.S.).
Vlodavets D; From the Department of Neurology, Boston Children's Hospital, Harvard Medical School, Boston (B.T.D.); the Developmental Neurology Unit, Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan (R.M., G.B.), and the Center of Translational and Experimental Myology, IRCCS Istituto Giannina Gaslini, Genoa (C.B.) - both in Italy; the Department of Developmental Neurology, Medical University of Gdańsk, Gdańsk, Poland (M.M.-B.); the Paediatric Gait Analysis Service of New South Wales, the Children's Hospital at Westmead and the University of Sydney, Sydney (K.R.); the Department of Pediatrics, Peking University First Hospital, Beijing (H.X.), and Children's Hospital of Fudan University, Shanghai (Y.W.) - both in China; the Department of Neurology, Faculdade de Medicina, Universidade de São Paulo, São Paulo (E.Z.); the Dubowitz Neuromuscular Centre, National Institute for Health Research Great Ormond Street Hospital Biomedical Research Centre, University College London Great Ormond Street Institute of Child Health, and Great Ormond Street Hospital for Children NHS Foundation Trust, London (G.B.), Roche Products, Welwyn Garden City (M.E.-K.), and the Muscular Dystrophy UK Oxford Neuromuscular Centre, the Department of Paediatrics, University of Oxford, Oxford (L.S.) - all in the United Kingdom; Russian Children Neuromuscular Center, Veltischev Clinical Pediatric Research Institute, Pirogov Russian National Research Medical University, Moscow (D.V.); Pharma Development, Safety (M.G.), Product Development Medical Affairs - Neuroscience and Rare Disease (K.G., P.F.), and Pharma Development Neurology (R.S.S.), F. Hoffmann-La Roche, and Roche Pharmaceutical Research and Early Development, Roche Innovation Center Basel (O.K., H.K.) - both in Basel, Switzerland; the Division of Child Neurology, Centre de Références des Maladies Neuromusculaires, the Department of Pediatrics, University Hospital Liege, University of Liege, Liege, Belgium (L.S.); and I-Motion, Institut de Myologie, Assistance Publique Hôpitaux de Paris, Hôpital Armand Trousseau, Paris (L.S.).
Wang Y; From the Department of Neurology, Boston Children's Hospital, Harvard Medical School, Boston (B.T.D.); the Developmental Neurology Unit, Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan (R.M., G.B.), and the Center of Translational and Experimental Myology, IRCCS Istituto Giannina Gaslini, Genoa (C.B.) - both in Italy; the Department of Developmental Neurology, Medical University of Gdańsk, Gdańsk, Poland (M.M.-B.); the Paediatric Gait Analysis Service of New South Wales, the Children's Hospital at Westmead and the University of Sydney, Sydney (K.R.); the Department of Pediatrics, Peking University First Hospital, Beijing (H.X.), and Children's Hospital of Fudan University, Shanghai (Y.W.) - both in China; the Department of Neurology, Faculdade de Medicina, Universidade de São Paulo, São Paulo (E.Z.); the Dubowitz Neuromuscular Centre, National Institute for Health Research Great Ormond Street Hospital Biomedical Research Centre, University College London Great Ormond Street Institute of Child Health, and Great Ormond Street Hospital for Children NHS Foundation Trust, London (G.B.), Roche Products, Welwyn Garden City (M.E.-K.), and the Muscular Dystrophy UK Oxford Neuromuscular Centre, the Department of Paediatrics, University of Oxford, Oxford (L.S.) - all in the United Kingdom; Russian Children Neuromuscular Center, Veltischev Clinical Pediatric Research Institute, Pirogov Russian National Research Medical University, Moscow (D.V.); Pharma Development, Safety (M.G.), Product Development Medical Affairs - Neuroscience and Rare Disease (K.G., P.F.), and Pharma Development Neurology (R.S.S.), F. Hoffmann-La Roche, and Roche Pharmaceutical Research and Early Development, Roche Innovation Center Basel (O.K., H.K.) - both in Basel, Switzerland; the Division of Child Neurology, Centre de Références des Maladies Neuromusculaires, the Department of Pediatrics, University Hospital Liege, University of Liege, Liege, Belgium (L.S.); and I-Motion, Institut de Myologie, Assistance Publique Hôpitaux de Paris, Hôpital Armand Trousseau, Paris (L.S.).
El-Khairi M; From the Department of Neurology, Boston Children's Hospital, Harvard Medical School, Boston (B.T.D.); the Developmental Neurology Unit, Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan (R.M., G.B.), and the Center of Translational and Experimental Myology, IRCCS Istituto Giannina Gaslini, Genoa (C.B.) - both in Italy; the Department of Developmental Neurology, Medical University of Gdańsk, Gdańsk, Poland (M.M.-B.); the Paediatric Gait Analysis Service of New South Wales, the Children's Hospital at Westmead and the University of Sydney, Sydney (K.R.); the Department of Pediatrics, Peking University First Hospital, Beijing (H.X.), and Children's Hospital of Fudan University, Shanghai (Y.W.) - both in China; the Department of Neurology, Faculdade de Medicina, Universidade de São Paulo, São Paulo (E.Z.); the Dubowitz Neuromuscular Centre, National Institute for Health Research Great Ormond Street Hospital Biomedical Research Centre, University College London Great Ormond Street Institute of Child Health, and Great Ormond Street Hospital for Children NHS Foundation Trust, London (G.B.), Roche Products, Welwyn Garden City (M.E.-K.), and the Muscular Dystrophy UK Oxford Neuromuscular Centre, the Department of Paediatrics, University of Oxford, Oxford (L.S.) - all in the United Kingdom; Russian Children Neuromuscular Center, Veltischev Clinical Pediatric Research Institute, Pirogov Russian National Research Medical University, Moscow (D.V.); Pharma Development, Safety (M.G.), Product Development Medical Affairs - Neuroscience and Rare Disease (K.G., P.F.), and Pharma Development Neurology (R.S.S.), F. Hoffmann-La Roche, and Roche Pharmaceutical Research and Early Development, Roche Innovation Center Basel (O.K., H.K.) - both in Basel, Switzerland; the Division of Child Neurology, Centre de Références des Maladies Neuromusculaires, the Department of Pediatrics, University Hospital Liege, University of Liege, Liege, Belgium (L.S.); and I-Motion, Institut de Myologie, Assistance Publique Hôpitaux de Paris, Hôpital Armand Trousseau, Paris (L.S.).
Gerber M; From the Department of Neurology, Boston Children's Hospital, Harvard Medical School, Boston (B.T.D.); the Developmental Neurology Unit, Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan (R.M., G.B.), and the Center of Translational and Experimental Myology, IRCCS Istituto Giannina Gaslini, Genoa (C.B.) - both in Italy; the Department of Developmental Neurology, Medical University of Gdańsk, Gdańsk, Poland (M.M.-B.); the Paediatric Gait Analysis Service of New South Wales, the Children's Hospital at Westmead and the University of Sydney, Sydney (K.R.); the Department of Pediatrics, Peking University First Hospital, Beijing (H.X.), and Children's Hospital of Fudan University, Shanghai (Y.W.) - both in China; the Department of Neurology, Faculdade de Medicina, Universidade de São Paulo, São Paulo (E.Z.); the Dubowitz Neuromuscular Centre, National Institute for Health Research Great Ormond Street Hospital Biomedical Research Centre, University College London Great Ormond Street Institute of Child Health, and Great Ormond Street Hospital for Children NHS Foundation Trust, London (G.B.), Roche Products, Welwyn Garden City (M.E.-K.), and the Muscular Dystrophy UK Oxford Neuromuscular Centre, the Department of Paediatrics, University of Oxford, Oxford (L.S.) - all in the United Kingdom; Russian Children Neuromuscular Center, Veltischev Clinical Pediatric Research Institute, Pirogov Russian National Research Medical University, Moscow (D.V.); Pharma Development, Safety (M.G.), Product Development Medical Affairs - Neuroscience and Rare Disease (K.G., P.F.), and Pharma Development Neurology (R.S.S.), F. Hoffmann-La Roche, and Roche Pharmaceutical Research and Early Development, Roche Innovation Center Basel (O.K., H.K.) - both in Basel, Switzerland; the Division of Child Neurology, Centre de Références des Maladies Neuromusculaires, the Department of Pediatrics, University Hospital Liege, University of Liege, Liege, Belgium (L.S.); and I-Motion, Institut de Myologie, Assistance Publique Hôpitaux de Paris, Hôpital Armand Trousseau, Paris (L.S.).
Gorni K; From the Department of Neurology, Boston Children's Hospital, Harvard Medical School, Boston (B.T.D.); the Developmental Neurology Unit, Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan (R.M., G.B.), and the Center of Translational and Experimental Myology, IRCCS Istituto Giannina Gaslini, Genoa (C.B.) - both in Italy; the Department of Developmental Neurology, Medical University of Gdańsk, Gdańsk, Poland (M.M.-B.); the Paediatric Gait Analysis Service of New South Wales, the Children's Hospital at Westmead and the University of Sydney, Sydney (K.R.); the Department of Pediatrics, Peking University First Hospital, Beijing (H.X.), and Children's Hospital of Fudan University, Shanghai (Y.W.) - both in China; the Department of Neurology, Faculdade de Medicina, Universidade de São Paulo, São Paulo (E.Z.); the Dubowitz Neuromuscular Centre, National Institute for Health Research Great Ormond Street Hospital Biomedical Research Centre, University College London Great Ormond Street Institute of Child Health, and Great Ormond Street Hospital for Children NHS Foundation Trust, London (G.B.), Roche Products, Welwyn Garden City (M.E.-K.), and the Muscular Dystrophy UK Oxford Neuromuscular Centre, the Department of Paediatrics, University of Oxford, Oxford (L.S.) - all in the United Kingdom; Russian Children Neuromuscular Center, Veltischev Clinical Pediatric Research Institute, Pirogov Russian National Research Medical University, Moscow (D.V.); Pharma Development, Safety (M.G.), Product Development Medical Affairs - Neuroscience and Rare Disease (K.G., P.F.), and Pharma Development Neurology (R.S.S.), F. Hoffmann-La Roche, and Roche Pharmaceutical Research and Early Development, Roche Innovation Center Basel (O.K., H.K.) - both in Basel, Switzerland; the Division of Child Neurology, Centre de Références des Maladies Neuromusculaires, the Department of Pediatrics, University Hospital Liege, University of Liege, Liege, Belgium (L.S.); and I-Motion, Institut de Myologie, Assistance Publique Hôpitaux de Paris, Hôpital Armand Trousseau, Paris (L.S.).
Khwaja O; From the Department of Neurology, Boston Children's Hospital, Harvard Medical School, Boston (B.T.D.); the Developmental Neurology Unit, Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan (R.M., G.B.), and the Center of Translational and Experimental Myology, IRCCS Istituto Giannina Gaslini, Genoa (C.B.) - both in Italy; the Department of Developmental Neurology, Medical University of Gdańsk, Gdańsk, Poland (M.M.-B.); the Paediatric Gait Analysis Service of New South Wales, the Children's Hospital at Westmead and the University of Sydney, Sydney (K.R.); the Department of Pediatrics, Peking University First Hospital, Beijing (H.X.), and Children's Hospital of Fudan University, Shanghai (Y.W.) - both in China; the Department of Neurology, Faculdade de Medicina, Universidade de São Paulo, São Paulo (E.Z.); the Dubowitz Neuromuscular Centre, National Institute for Health Research Great Ormond Street Hospital Biomedical Research Centre, University College London Great Ormond Street Institute of Child Health, and Great Ormond Street Hospital for Children NHS Foundation Trust, London (G.B.), Roche Products, Welwyn Garden City (M.E.-K.), and the Muscular Dystrophy UK Oxford Neuromuscular Centre, the Department of Paediatrics, University of Oxford, Oxford (L.S.) - all in the United Kingdom; Russian Children Neuromuscular Center, Veltischev Clinical Pediatric Research Institute, Pirogov Russian National Research Medical University, Moscow (D.V.); Pharma Development, Safety (M.G.), Product Development Medical Affairs - Neuroscience and Rare Disease (K.G., P.F.), and Pharma Development Neurology (R.S.S.), F. Hoffmann-La Roche, and Roche Pharmaceutical Research and Early Development, Roche Innovation Center Basel (O.K., H.K.) - both in Basel, Switzerland; the Division of Child Neurology, Centre de Références des Maladies Neuromusculaires, the Department of Pediatrics, University Hospital Liege, University of Liege, Liege, Belgium (L.S.); and I-Motion, Institut de Myologie, Assistance Publique Hôpitaux de Paris, Hôpital Armand Trousseau, Paris (L.S.).
Kletzl H; From the Department of Neurology, Boston Children's Hospital, Harvard Medical School, Boston (B.T.D.); the Developmental Neurology Unit, Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan (R.M., G.B.), and the Center of Translational and Experimental Myology, IRCCS Istituto Giannina Gaslini, Genoa (C.B.) - both in Italy; the Department of Developmental Neurology, Medical University of Gdańsk, Gdańsk, Poland (M.M.-B.); the Paediatric Gait Analysis Service of New South Wales, the Children's Hospital at Westmead and the University of Sydney, Sydney (K.R.); the Department of Pediatrics, Peking University First Hospital, Beijing (H.X.), and Children's Hospital of Fudan University, Shanghai (Y.W.) - both in China; the Department of Neurology, Faculdade de Medicina, Universidade de São Paulo, São Paulo (E.Z.); the Dubowitz Neuromuscular Centre, National Institute for Health Research Great Ormond Street Hospital Biomedical Research Centre, University College London Great Ormond Street Institute of Child Health, and Great Ormond Street Hospital for Children NHS Foundation Trust, London (G.B.), Roche Products, Welwyn Garden City (M.E.-K.), and the Muscular Dystrophy UK Oxford Neuromuscular Centre, the Department of Paediatrics, University of Oxford, Oxford (L.S.) - all in the United Kingdom; Russian Children Neuromuscular Center, Veltischev Clinical Pediatric Research Institute, Pirogov Russian National Research Medical University, Moscow (D.V.); Pharma Development, Safety (M.G.), Product Development Medical Affairs - Neuroscience and Rare Disease (K.G., P.F.), and Pharma Development Neurology (R.S.S.), F. Hoffmann-La Roche, and Roche Pharmaceutical Research and Early Development, Roche Innovation Center Basel (O.K., H.K.) - both in Basel, Switzerland; the Division of Child Neurology, Centre de Références des Maladies Neuromusculaires, the Department of Pediatrics, University Hospital Liege, University of Liege, Liege, Belgium (L.S.); and I-Motion, Institut de Myologie, Assistance Publique Hôpitaux de Paris, Hôpital Armand Trousseau, Paris (L.S.).
Scalco RS; From the Department of Neurology, Boston Children's Hospital, Harvard Medical School, Boston (B.T.D.); the Developmental Neurology Unit, Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan (R.M., G.B.), and the Center of Translational and Experimental Myology, IRCCS Istituto Giannina Gaslini, Genoa (C.B.) - both in Italy; the Department of Developmental Neurology, Medical University of Gdańsk, Gdańsk, Poland (M.M.-B.); the Paediatric Gait Analysis Service of New South Wales, the Children's Hospital at Westmead and the University of Sydney, Sydney (K.R.); the Department of Pediatrics, Peking University First Hospital, Beijing (H.X.), and Children's Hospital of Fudan University, Shanghai (Y.W.) - both in China; the Department of Neurology, Faculdade de Medicina, Universidade de São Paulo, São Paulo (E.Z.); the Dubowitz Neuromuscular Centre, National Institute for Health Research Great Ormond Street Hospital Biomedical Research Centre, University College London Great Ormond Street Institute of Child Health, and Great Ormond Street Hospital for Children NHS Foundation Trust, London (G.B.), Roche Products, Welwyn Garden City (M.E.-K.), and the Muscular Dystrophy UK Oxford Neuromuscular Centre, the Department of Paediatrics, University of Oxford, Oxford (L.S.) - all in the United Kingdom; Russian Children Neuromuscular Center, Veltischev Clinical Pediatric Research Institute, Pirogov Russian National Research Medical University, Moscow (D.V.); Pharma Development, Safety (M.G.), Product Development Medical Affairs - Neuroscience and Rare Disease (K.G., P.F.), and Pharma Development Neurology (R.S.S.), F. Hoffmann-La Roche, and Roche Pharmaceutical Research and Early Development, Roche Innovation Center Basel (O.K., H.K.) - both in Basel, Switzerland; the Division of Child Neurology, Centre de Références des Maladies Neuromusculaires, the Department of Pediatrics, University Hospital Liege, University of Liege, Liege, Belgium (L.S.); and I-Motion, Institut de Myologie, Assistance Publique Hôpitaux de Paris, Hôpital Armand Trousseau, Paris (L.S.).
Fontoura P; From the Department of Neurology, Boston Children's Hospital, Harvard Medical School, Boston (B.T.D.); the Developmental Neurology Unit, Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan (R.M., G.B.), and the Center of Translational and Experimental Myology, IRCCS Istituto Giannina Gaslini, Genoa (C.B.) - both in Italy; the Department of Developmental Neurology, Medical University of Gdańsk, Gdańsk, Poland (M.M.-B.); the Paediatric Gait Analysis Service of New South Wales, the Children's Hospital at Westmead and the University of Sydney, Sydney (K.R.); the Department of Pediatrics, Peking University First Hospital, Beijing (H.X.), and Children's Hospital of Fudan University, Shanghai (Y.W.) - both in China; the Department of Neurology, Faculdade de Medicina, Universidade de São Paulo, São Paulo (E.Z.); the Dubowitz Neuromuscular Centre, National Institute for Health Research Great Ormond Street Hospital Biomedical Research Centre, University College London Great Ormond Street Institute of Child Health, and Great Ormond Street Hospital for Children NHS Foundation Trust, London (G.B.), Roche Products, Welwyn Garden City (M.E.-K.), and the Muscular Dystrophy UK Oxford Neuromuscular Centre, the Department of Paediatrics, University of Oxford, Oxford (L.S.) - all in the United Kingdom; Russian Children Neuromuscular Center, Veltischev Clinical Pediatric Research Institute, Pirogov Russian National Research Medical University, Moscow (D.V.); Pharma Development, Safety (M.G.), Product Development Medical Affairs - Neuroscience and Rare Disease (K.G., P.F.), and Pharma Development Neurology (R.S.S.), F. Hoffmann-La Roche, and Roche Pharmaceutical Research and Early Development, Roche Innovation Center Basel (O.K., H.K.) - both in Basel, Switzerland; the Division of Child Neurology, Centre de Références des Maladies Neuromusculaires, the Department of Pediatrics, University Hospital Liege, University of Liege, Liege, Belgium (L.S.); and I-Motion, Institut de Myologie, Assistance Publique Hôpitaux de Paris, Hôpital Armand Trousseau, Paris (L.S.).
Servais L; From the Department of Neurology, Boston Children's Hospital, Harvard Medical School, Boston (B.T.D.); the Developmental Neurology Unit, Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan (R.M., G.B.), and the Center of Translational and Experimental Myology, IRCCS Istituto Giannina Gaslini, Genoa (C.B.) - both in Italy; the Department of Developmental Neurology, Medical University of Gdańsk, Gdańsk, Poland (M.M.-B.); the Paediatric Gait Analysis Service of New South Wales, the Children's Hospital at Westmead and the University of Sydney, Sydney (K.R.); the Department of Pediatrics, Peking University First Hospital, Beijing (H.X.), and Children's Hospital of Fudan University, Shanghai (Y.W.) - both in China; the Department of Neurology, Faculdade de Medicina, Universidade de São Paulo, São Paulo (E.Z.); the Dubowitz Neuromuscular Centre, National Institute for Health Research Great Ormond Street Hospital Biomedical Research Centre, University College London Great Ormond Street Institute of Child Health, and Great Ormond Street Hospital for Children NHS Foundation Trust, London (G.B.), Roche Products, Welwyn Garden City (M.E.-K.), and the Muscular Dystrophy UK Oxford Neuromuscular Centre, the Department of Paediatrics, University of Oxford, Oxford (L.S.) - all in the United Kingdom; Russian Children Neuromuscular Center, Veltischev Clinical Pediatric Research Institute, Pirogov Russian National Research Medical University, Moscow (D.V.); Pharma Development, Safety (M.G.), Product Development Medical Affairs - Neuroscience and Rare Disease (K.G., P.F.), and Pharma Development Neurology (R.S.S.), F. Hoffmann-La Roche, and Roche Pharmaceutical Research and Early Development, Roche Innovation Center Basel (O.K., H.K.) - both in Basel, Switzerland; the Division of Child Neurology, Centre de Références des Maladies Neuromusculaires, the Department of Pediatrics, University Hospital Liege, University of Liege, Liege, Belgium (L.S.); and I-Motion, Institut de Myologie, Assistance Publique Hôpitaux de Paris, Hôpital Armand Trousseau, Paris (L.S.).
Pokaż więcej
Corporate Authors :
FIREFISH Working Group
Źródło :
The New England journal of medicine [N Engl J Med] 2021 Jul 29; Vol. 385 (5), pp. 427-435.
Typ publikacji :
Journal Article; Research Support, Non-U.S. Gov't
MeSH Terms :
Azo Compounds/*therapeutic use
Neuromuscular Agents/*therapeutic use
Pyrimidines/*therapeutic use
Spinal Muscular Atrophies of Childhood/*drug therapy
Azo Compounds/adverse effects ; Female ; Historically Controlled Study ; Humans ; Infant ; Male ; Motor Skills/drug effects ; Neuromuscular Agents/adverse effects ; Progression-Free Survival ; Pyrimidines/adverse effects ; Severity of Illness Index ; Spinal Muscular Atrophies of Childhood/mortality ; Spinal Muscular Atrophies of Childhood/physiopathology
Czasopismo naukowe
Tytuł :
Changes in traumatic mechanisms of injury in Southern California related to COVID-19: Penetrating trauma as a second pandemic.
Autorzy :
Yeates EO; From the Department of Surgery (E.O.Y., A.G., C.Barrios., J.C., A.N., A. Leung., C.G., J.N.), University of California, Irvine, Orange; Department of Surgery (A.G., M.S., N.O.), University of Southern California; Department of Surgery (G.B., D.M.), Cedars-Sinai Medical Center; Department of Surgery (C.J., K.G., H.C., A.T.), University of California, Los Angeles, Los Angeles; Department of Surgery (S.B., L.P.-V., A. Lin., R.A.F.), Loma Linda University, Loma Linda; Department of Surgery (M.B.), Riverside/Riverside University Health System, University of California; Riverside University Health System (C.F.), Comparative Effectiveness and Clinical Outcomes Research Center, Moreno Valley; Department of Surgery (T. Costantini, J.S., T. Curry), University of California, San Diego; Department of Surgery (D.W.), Sharp Memorial Hospital, San Diego; Trauma Department (W.L.B., K.B.S.), Scripps Memorial Hospital La Jolla, La Jolla; Department of Surgery (T.K.D., C. Barbaro., G.D.), Ventura County Medical Center, Ventura; and Department of Trauma (A.J.), Santa Barbara Cottage Hospital, Santa Barbara, California.
Grigorian A
Barrios C
Schellenberg M
Owattanapanich N
Barmparas G
Margulies D
Juillard C
Garber K
Cryer H
Tillou A
Burruss S
Penaloza-Villalobos L
Lin A
Figueras RA
Brenner M
Firek C
Costantini T
Santorelli J
Curry T
Wintz D
Biffl WL
Schaffer KB
Duncan TK
Barbaro C
Diaz G
Johnson A
Chinn J
Naaseh A
Leung A
Grabar C
Nahmias J
Pokaż więcej
Źródło :
The journal of trauma and acute care surgery [J Trauma Acute Care Surg] 2021 Apr 01; Vol. 90 (4), pp. 714-721.
Typ publikacji :
Journal Article; Multicenter Study
MeSH Terms :
COVID-19*/epidemiology
COVID-19*/prevention & control
COVID-19*/psychology
Physical Distancing*
Domestic Violence/*statistics & numerical data
Suicide, Attempted/*statistics & numerical data
Wounds, Gunshot/*epidemiology
Wounds, Penetrating/*epidemiology
Adult ; California/epidemiology ; Female ; Historically Controlled Study ; Humans ; Male ; Retrospective Studies ; SARS-CoV-2
Czasopismo naukowe
Tytuł :
Feasibility and efficacy of modified fixed citrate concentration protocol using only commercial preparations in critically ill patients: a prospective cohort study with a historical control group.
Autorzy :
Shi Y; Department of medical ICU, Peking Union Medical College Hospital, Peking Union Medical College and Chinese Academy of Medical Sciences, 1 Shuai Fu Yuan, Dongcheng District, Beijing, 100730, China. .
Qin HY; Department of medical ICU, Peking Union Medical College Hospital, Peking Union Medical College and Chinese Academy of Medical Sciences, 1 Shuai Fu Yuan, Dongcheng District, Beijing, 100730, China.
Peng JM; Department of medical ICU, Peking Union Medical College Hospital, Peking Union Medical College and Chinese Academy of Medical Sciences, 1 Shuai Fu Yuan, Dongcheng District, Beijing, 100730, China.
Hu XY; Department of medical ICU, Peking Union Medical College Hospital, Peking Union Medical College and Chinese Academy of Medical Sciences, 1 Shuai Fu Yuan, Dongcheng District, Beijing, 100730, China.
Du B; Department of medical ICU, Peking Union Medical College Hospital, Peking Union Medical College and Chinese Academy of Medical Sciences, 1 Shuai Fu Yuan, Dongcheng District, Beijing, 100730, China.
Pokaż więcej
Źródło :
BMC anesthesiology [BMC Anesthesiol] 2021 Mar 30; Vol. 21 (1), pp. 96. Date of Electronic Publication: 2021 Mar 30.
Typ publikacji :
Journal Article
MeSH Terms :
Critical Illness*
Anticoagulants/*administration & dosage
Citric Acid/*administration & dosage
Calcium/blood ; Cohort Studies ; Continuous Renal Replacement Therapy ; Dose-Response Relationship, Drug ; Feasibility Studies ; Female ; Historically Controlled Study ; Humans ; Male ; Middle Aged
Czasopismo naukowe
Tytuł :
COVID-19-induced hyperinflammation, immunosuppression, recovery and survival: how causal inference may help draw robust conclusions.
Autorzy :
Landewé RBM; Amsterdam Rheumatology Center, AMC, Amsterdam, The Netherlands .; Rheumatology, Zuyderland MC, Heerlen, The Netherlands.
Ramiro S; Rheumatology, Leiden University Medical Center, Leiden, The Netherlands.; Rheumatology, Zuyderland Medical Center, Heerlen, The Netherlands.
Mostard RLM; Department of Pulmonology, Zuyderland Medical Centre Heerlen, Heerlen, The Netherlands.
Pokaż więcej
Źródło :
RMD open [RMD Open] 2021 Mar; Vol. 7 (1).
Typ publikacji :
Journal Article
MeSH Terms :
Antibodies, Monoclonal, Humanized/*therapeutic use
C-Reactive Protein/*immunology
COVID-19/*drug therapy
Cytokine Release Syndrome/*drug therapy
Glucocorticoids/*therapeutic use
Immunosuppressive Agents/*therapeutic use
Methylprednisolone/*therapeutic use
COVID-19/immunology ; Causality ; Cytokine Release Syndrome/immunology ; Historically Controlled Study ; Humans ; Inflammation/immunology ; Mediation Analysis ; Mortality ; SARS-CoV-2 ; Survival Rate ; Treatment Outcome
SCR Protocol :
COVID-19 drug treatment
Czasopismo naukowe
Tytuł :
Meropenem versus piperacillin/tazobactam for febrile neutropenia in pediatric patients: efficacy of piperacillin/tazobactam as a 1-h drip infusion four times a day.
Autorzy :
Kobayashi R; Department of Hematology/Oncology for Children and Adolescents, Sapporo Hokuyu Hospital, 6-6 Higashi-Sapporo, Shiroishiku, Sapporo, 003-0006, Japan. .
Sano H; Department of Hematology/Oncology for Children and Adolescents, Sapporo Hokuyu Hospital, 6-6 Higashi-Sapporo, Shiroishiku, Sapporo, 003-0006, Japan.
Matsushima S; Department of Hematology/Oncology for Children and Adolescents, Sapporo Hokuyu Hospital, 6-6 Higashi-Sapporo, Shiroishiku, Sapporo, 003-0006, Japan.
Hori D; Department of Hematology/Oncology for Children and Adolescents, Sapporo Hokuyu Hospital, 6-6 Higashi-Sapporo, Shiroishiku, Sapporo, 003-0006, Japan.
Yanagi M; Department of Hematology/Oncology for Children and Adolescents, Sapporo Hokuyu Hospital, 6-6 Higashi-Sapporo, Shiroishiku, Sapporo, 003-0006, Japan.
Kodama K; Department of Hematology/Oncology for Children and Adolescents, Sapporo Hokuyu Hospital, 6-6 Higashi-Sapporo, Shiroishiku, Sapporo, 003-0006, Japan.
Suzuki D; Department of Hematology/Oncology for Children and Adolescents, Sapporo Hokuyu Hospital, 6-6 Higashi-Sapporo, Shiroishiku, Sapporo, 003-0006, Japan.
Kobayashi K; Department of Hematology/Oncology for Children and Adolescents, Sapporo Hokuyu Hospital, 6-6 Higashi-Sapporo, Shiroishiku, Sapporo, 003-0006, Japan.
Pokaż więcej
Źródło :
International journal of hematology [Int J Hematol] 2021 Mar; Vol. 113 (3), pp. 430-435. Date of Electronic Publication: 2020 Nov 10.
Typ publikacji :
Comparative Study; Equivalence Trial; Journal Article
MeSH Terms :
Anti-Bacterial Agents/*therapeutic use
Bacteremia/*drug therapy
Febrile Neutropenia/*drug therapy
Meropenem/*therapeutic use
Piperacillin, Tazobactam Drug Combination/*therapeutic use
Adolescent ; Adult ; Anti-Bacterial Agents/administration & dosage ; Bacteremia/etiology ; Body Weight ; Child ; Child, Preschool ; Drug Administration Schedule ; Drug Therapy, Combination ; Febrile Neutropenia/complications ; Historically Controlled Study ; Humans ; Immunologic Deficiency Syndromes/drug therapy ; Immunologic Deficiency Syndromes/therapy ; Infant ; Infant, Newborn ; Infusions, Intravenous ; Maximum Tolerated Dose ; Meropenem/administration & dosage ; Neoplasms/drug therapy ; Neoplasms/therapy ; Piperacillin, Tazobactam Drug Combination/administration & dosage ; Stem Cell Transplantation ; Young Adult
Czasopismo naukowe
Tytuł :
A regimen with caplacizumab, immunosuppression, and plasma exchange prevents unfavorable outcomes in immune-mediated TTP.
Autorzy :
Coppo P; Centre de Référence des Microangiopathies Thrombotiques, Assistance Publique-Hôpitaux de Paris (AP-HP), Paris, France.; Service d'Hématologie, APHP Sorbonne Université, Paris, France.; INSERM Unité Mixte de Recherche S (UMRS) 1138, Centre de Recherche des Cordeliers, Paris, France.
Bubenheim M; Department of Clinical Research and Innovation, Centre Hospitalier Universitaire (CHU) Rouen, Rouen, France.
Azoulay E; Centre de Référence des Microangiopathies Thrombotiques, Assistance Publique-Hôpitaux de Paris (AP-HP), Paris, France.; Médecine Intensive Réanimation, Hôpital Saint Louis, AP-HP, Paris, France.; Paris University, Paris, France.
Galicier L; Centre de Référence des Microangiopathies Thrombotiques, Assistance Publique-Hôpitaux de Paris (AP-HP), Paris, France.; Paris University, Paris, France.; Service d'Immunologie Clinique, Hôpital Saint-Louis, Assistance Publique-Hôpitaux de Paris, Paris, France.
Malot S; Centre de Référence des Microangiopathies Thrombotiques, Assistance Publique-Hôpitaux de Paris (AP-HP), Paris, France.
Bigé N; Centre de Référence des Microangiopathies Thrombotiques, Assistance Publique-Hôpitaux de Paris (AP-HP), Paris, France.; Service de Médecine Intensive et Réanimation, Hôpital Saint-Antoine, AP-HP, Paris, France.
Poullin P; Centre de Référence des Microangiopathies Thrombotiques, Assistance Publique-Hôpitaux de Paris (AP-HP), Paris, France.; Service d'Hémaphérèse, CHU La Timone, Marseille, France.
Provôt F; Centre de Référence des Microangiopathies Thrombotiques, Assistance Publique-Hôpitaux de Paris (AP-HP), Paris, France.; Service de Néphrologie, Hôpital Albert Calmette, Lille, France.
Martis N; Service de Médecine Interne, CHU de Nice, Nice, France.
Presne C; Centre de Référence des Microangiopathies Thrombotiques, Assistance Publique-Hôpitaux de Paris (AP-HP), Paris, France.; Service de Néphrologie, Hôpital Sud, CHU Amiens, Amiens, France.
Moranne O; Service de Néphrologie-Dialyse-Aphérèse, Hôpital Universitaire de Nîmes, Nimes, France.
Benainous R; Service de Médecine Interne, CHU Avicenne, Bobigny, France.
Dossier A; Service de Médecine Interne, CHU Bichat, Paris, France.
Seguin A; Centre de Référence des Microangiopathies Thrombotiques, Assistance Publique-Hôpitaux de Paris (AP-HP), Paris, France.; Service de Réanimation Médicale, CHU de Nantes, Nantes, France.
Hié M; Centre de Référence des Microangiopathies Thrombotiques, Assistance Publique-Hôpitaux de Paris (AP-HP), Paris, France.; Service de Médecine Interne, CHU La Pitié Salpétrière, Paris, France.
Wynckel A; Centre de Référence des Microangiopathies Thrombotiques, Assistance Publique-Hôpitaux de Paris (AP-HP), Paris, France.; Service de Néphrologie, Hôpital Maison Blanche, Reims, France.
Delmas Y; Centre de Référence des Microangiopathies Thrombotiques, Assistance Publique-Hôpitaux de Paris (AP-HP), Paris, France.; Service de Néphrologie, CHU Bordeaux, Bordeaux, France.
Augusto JF; Centre de Référence des Microangiopathies Thrombotiques, Assistance Publique-Hôpitaux de Paris (AP-HP), Paris, France.; Service de Néphrologie-Dialyse-Transplantation, CHU d'Angers, d'Angers, France.
Perez P; Centre de Référence des Microangiopathies Thrombotiques, Assistance Publique-Hôpitaux de Paris (AP-HP), Paris, France.; Service de Médecine Intensive Réanimation, Hôpital Brabois, Nancy, France.
Rieu V; Centre de Référence des Microangiopathies Thrombotiques, Assistance Publique-Hôpitaux de Paris (AP-HP), Paris, France.; Service de Médecine Interne, CHU de Clermont-Ferrand, Clermont-Ferrand, France.
Barbet C; Centre de Référence des Microangiopathies Thrombotiques, Assistance Publique-Hôpitaux de Paris (AP-HP), Paris, France.; Service de Néphrologie-Immunologie Clinique, CHRU de Tours, Tours, France.
Lhote F; Service de Médecine Interne, CH Delafontaine, Saint-Denis, France.
Ulrich M; Service de Néphrologie, CH de Valenciennes, Valenciennes, France.
Rumpler AC; Centre de Référence des Microangiopathies Thrombotiques, Assistance Publique-Hôpitaux de Paris (AP-HP), Paris, France.; Service d'Hématologie, CHRU de Besançon, Besançon, France.
de Witte S; Service d'Hématologie, Centre Hospitalier de Libourne, Libourne, France.
Krummel T; Centre de Référence des Microangiopathies Thrombotiques, Assistance Publique-Hôpitaux de Paris (AP-HP), Paris, France.; Service de Néphrologie, Centre Hospitalier Régional Universitaire de Strasbourg, Strasbourg, France.
Veyradier A; Centre de Référence des Microangiopathies Thrombotiques, Assistance Publique-Hôpitaux de Paris (AP-HP), Paris, France.; Hématologie Biologique, Hôpital Lariboisière, AP-HP, Paris, France.; Équipe d'accueil-3518, Institut de Recherche Saint Louis, Université de Paris, Paris, France.
Benhamou Y; Centre de Référence des Microangiopathies Thrombotiques, Assistance Publique-Hôpitaux de Paris (AP-HP), Paris, France.; Département de Médecine Interne, CHU Charles Nicolle, Rouen, France; and.; Normandie University, UNIROUEN, INSERM U1096 EnVI, Rouen, France.
Pokaż więcej
Źródło :
Blood [Blood] 2021 Feb 11; Vol. 137 (6), pp. 733-742.
Typ publikacji :
Journal Article; Multicenter Study; Research Support, Non-U.S. Gov't
MeSH Terms :
Plasma Exchange*
Adrenal Cortex Hormones/*therapeutic use
Purpura, Thrombotic Thrombocytopenic/*therapy
Rituximab/*therapeutic use
Single-Domain Antibodies/*therapeutic use
ADAMTS13 Protein/blood ; Adult ; Combined Modality Therapy ; Compassionate Use Trials ; Disease Progression ; Drug Therapy, Combination ; Female ; Hemorrhage/chemically induced ; Historically Controlled Study ; Humans ; Male ; Middle Aged ; Platelet Count ; Prospective Studies ; Purpura, Thrombotic Thrombocytopenic/blood ; Purpura, Thrombotic Thrombocytopenic/drug therapy ; Purpura, Thrombotic Thrombocytopenic/mortality ; Severity of Illness Index ; Single-Domain Antibodies/adverse effects ; Single-Domain Antibodies/economics ; Thromboembolism/etiology ; Treatment Outcome ; von Willebrand Factor/antagonists & inhibitors
Czasopismo naukowe
Tytuł :
Use of a historical control group in a noninferiority trial assessing a new antibacterial treatment: A case study and discussion of practical implementation aspects.
Autorzy :
Dejardin D; Department of Biostatistics, F. Hoffmann-La Roche, Basel, Switzerland.
Delmar P; Department of Biostatistics, F. Hoffmann-La Roche, Basel, Switzerland.
Warne C; Department of Biostatistics, Roche Products Ltd, Welwyn Garden City, UK.
Patel K; Department of Biostatistics, Roche Products Ltd, Welwyn Garden City, UK.
van Rosmalen J; Department of Biostatistics, Erasmus University Medical Center, Rotterdam, the Netherlands.
Lesaffre E; Interuniversity Institute for Biostatistics and Statistical Bioinformatics, KU Leuven, Leuven, Belgium.
Pokaż więcej
Źródło :
Pharmaceutical statistics [Pharm Stat] 2018 Mar; Vol. 17 (2), pp. 169-181. Date of Electronic Publication: 2017 Dec 28.
Typ publikacji :
Journal Article
MeSH Terms :
Computer Simulation*/statistics & numerical data
Anti-Bacterial Agents/*therapeutic use
Healthcare-Associated Pneumonia/*drug therapy
Historically Controlled Study/*methods
Pneumonia, Ventilator-Associated/*drug therapy
Bayes Theorem ; Healthcare-Associated Pneumonia/epidemiology ; Historically Controlled Study/statistics & numerical data ; Humans ; Pneumonia, Ventilator-Associated/epidemiology ; Sample Size ; Treatment Outcome
Czasopismo naukowe
Tytuł :
The traditional experience strategy (TES) and combined ultrasonography examination (CUE) for the treatment of lateral compression type 1 pelvic fractures: a historical control study.
Autorzy :
Huang H; Department of Orthopedic Trauma, Honghui Hospital, Xi'an Jiaotong University, Beilin District, No. 555 Youyi East Road, Shaanxi Province, 710054, Xi'an, Republic of China.
Zhang BF; Department of Orthopedic Trauma, Honghui Hospital, Xi'an Jiaotong University, Beilin District, No. 555 Youyi East Road, Shaanxi Province, 710054, Xi'an, Republic of China.
Liu P; Department of Orthopedic Trauma, Honghui Hospital, Xi'an Jiaotong University, Beilin District, No. 555 Youyi East Road, Shaanxi Province, 710054, Xi'an, Republic of China.
Deng HL; Department of Orthopedic Trauma, Honghui Hospital, Xi'an Jiaotong University, Beilin District, No. 555 Youyi East Road, Shaanxi Province, 710054, Xi'an, Republic of China.
Wang PF; Department of Orthopedic Trauma, Honghui Hospital, Xi'an Jiaotong University, Beilin District, No. 555 Youyi East Road, Shaanxi Province, 710054, Xi'an, Republic of China.
Wang H; Department of Orthopedic Trauma, Honghui Hospital, Xi'an Jiaotong University, Beilin District, No. 555 Youyi East Road, Shaanxi Province, 710054, Xi'an, Republic of China.
Li BF; Department of Orthopedic Trauma, Honghui Hospital, Xi'an Jiaotong University, Beilin District, No. 555 Youyi East Road, Shaanxi Province, 710054, Xi'an, Republic of China.
Cong YX; Department of Orthopedic Trauma, Honghui Hospital, Xi'an Jiaotong University, Beilin District, No. 555 Youyi East Road, Shaanxi Province, 710054, Xi'an, Republic of China. .
Zhuang Y; Department of Orthopedic Trauma, Honghui Hospital, Xi'an Jiaotong University, Beilin District, No. 555 Youyi East Road, Shaanxi Province, 710054, Xi'an, Republic of China. .
Pokaż więcej
Źródło :
BMC musculoskeletal disorders [BMC Musculoskelet Disord] 2021 Jan 25; Vol. 22 (1), pp. 110. Date of Electronic Publication: 2021 Jan 25.
Typ publikacji :
Journal Article
MeSH Terms :
Fractures, Bone*/diagnostic imaging
Fractures, Bone*/surgery
Fractures, Compression*
Pelvic Bones*/diagnostic imaging
Pelvic Bones*/surgery
Fracture Fixation, Internal ; Historically Controlled Study ; Humans ; Pelvis ; Retrospective Studies ; Treatment Outcome ; Ultrasonography
Czasopismo naukowe
Tytuł :
Combination Therapy Using Prothrombin Complex Concentrate and Vitamin K in Anticoagulated Patients with Traumatic Intracranial Hemorrhage Prevents Progressive Hemorrhagic Injury: A Historically Controlled Study.
Autorzy :
Koyama H; Department of Neurosurgery, Kawasaki Medical School.; Department of Neurosurgery, Tokushima University Hospital.
Yagi K; Department of Neurosurgery, Kawasaki Medical School.
Hara K; Department of Neurosurgery, Kawasaki Medical School.
Matsubara S; Department of Neurosurgery, Kawasaki Medical School.
Tao Y; Department of Neurosurgery, Kawasaki Medical School.
Uno M; Department of Neurosurgery, Kawasaki Medical School.
Pokaż więcej
Źródło :
Neurologia medico-chirurgica [Neurol Med Chir (Tokyo)] 2021 Jan 15; Vol. 61 (1), pp. 47-54. Date of Electronic Publication: 2020 Nov 19.
Typ publikacji :
Journal Article
MeSH Terms :
Anticoagulants/*adverse effects
Blood Coagulation/*drug effects
Blood Coagulation Factors/*therapeutic use
Intracranial Hemorrhage, Traumatic/*drug therapy
Vitamin K/*therapeutic use
Warfarin/*adverse effects
Warfarin/*antagonists & inhibitors
Aged ; Aged, 80 and over ; Anticoagulants/therapeutic use ; Drug Therapy, Combination ; Female ; Historically Controlled Study ; Humans ; Japan ; Male ; Retrospective Studies ; Warfarin/therapeutic use
Czasopismo naukowe
Tytuł :
Prospective, historically controlled study to evaluate the efficacy and safety of a new paediatric formulation of nifurtimox in children aged 0 to 17 years with Chagas disease one year after treatment (CHICO).
Autorzy :
Altcheh J; Hospital de Niños Ricardo Gutiérrez and Instituto Multidisciplinario de Investigacion en Patologias Pediatricas (IMIPP), CONICET-GCBA, Buenos Aires, Argentina.
Castro L; Centro de Atencion e Investigacion Medica S.A. (CAIMED-Yopal), Yopal, Colombia.
Dib JC; Universidad del Norte, Barranquilla, Colombia.
Grossmann U; Bayer AG, Research and Development Pharmaceuticals, Berlin, Germany.
Huang E; Bayer US LLC, Whippany, New Jersey, United States of America.
Moscatelli G; Hospital de Niños Ricardo Gutiérrez and Instituto Multidisciplinario de Investigacion en Patologias Pediatricas (IMIPP), CONICET-GCBA, Buenos Aires, Argentina.
Pinto Rocha JJ; Fundación CEADES-Plataforma de Atención Integral a los Pacientes con Enfermedades de Chagas, Cochabamba, Bolivia.
Ramírez TE; Centro de Enfermedad de Chagas y Patologias Regionales, Santiago del Estero, Argentina.
Pokaż więcej
Corporate Authors :
CHICO Study Group
Źródło :
PLoS neglected tropical diseases [PLoS Negl Trop Dis] 2021 Jan 07; Vol. 15 (1), pp. e0008912. Date of Electronic Publication: 2021 Jan 07 (Print Publication: 2021).
Typ publikacji :
Clinical Trial, Phase III; Journal Article; Randomized Controlled Trial; Research Support, Non-U.S. Gov't
MeSH Terms :
Chagas Disease/*drug therapy
Nifurtimox/*therapeutic use
Adolescent ; Chagas Disease/diagnosis ; Child ; Child, Preschool ; Female ; Historically Controlled Study ; Humans ; Infant ; Infant, Newborn ; Male ; Prospective Studies ; South America ; Treatment Outcome ; Trypanocidal Agents/therapeutic use ; Trypanosoma cruzi/immunology
Czasopismo naukowe
Tytuł :
Designing historical control studies with survival endpoints using exact statistical inference.
Autorzy :
Han G; Department of Epidemiology and Biostatistics, School of Public Health, Texas A&M University, College Station, Texas, USA.
Pokaż więcej
Źródło :
Pharmaceutical statistics [Pharm Stat] 2021 Jan; Vol. 20 (1), pp. 4-14. Date of Electronic Publication: 2020 Aug 03.
Typ publikacji :
Journal Article; Research Support, Non-U.S. Gov't
MeSH Terms :
Models, Statistical*
Research Design*
Bias ; Historically Controlled Study ; Humans ; Sample Size
Czasopismo naukowe
Tytuł :
An enhanced recovery programme improves the comfort and outcomes in children with obstructive sleep apnoea undergoing adenotonsillectomy: A retrospective historical control study.
Autorzy :
Zhang Y; Department of Otolaryngology, Head and Neck Surgery, Yantai Yuhuangding Hospital, Qingdao University, Yantai, China.
Liu D; Department of Otolaryngology, Head and Neck Surgery, Yantai Yuhuangding Hospital, Qingdao University, Yantai, China.
Chen X; Department of Otolaryngology, Head and Neck Surgery, Yantai Yuhuangding Hospital, Qingdao University, Yantai, China.
Ma J; Department of Anesthesiology. Yantai Yuhuangding Hospital, Qingdao University, Yantai, China.
Song X; Department of Otolaryngology, Head and Neck Surgery, Yantai Yuhuangding Hospital, Qingdao University, Yantai, China.
Pokaż więcej
Źródło :
Clinical otolaryngology : official journal of ENT-UK ; official journal of Netherlands Society for Oto-Rhino-Laryngology & Cervico-Facial Surgery [Clin Otolaryngol] 2021 Jan; Vol. 46 (1), pp. 249-255. Date of Electronic Publication: 2020 Nov 07.
Typ publikacji :
Journal Article
MeSH Terms :
Adenoidectomy*
Enhanced Recovery After Surgery*
Tonsillectomy*
Sleep Apnea, Obstructive/*surgery
Adolescent ; Child ; Child, Preschool ; Female ; Historically Controlled Study ; Humans ; Male ; Patient Comfort ; Retrospective Studies ; Treatment Outcome
Czasopismo naukowe
Tytuł :
Assessment of respiratory muscles and motor function in children with SMA treated by nusinersen.
Autorzy :
Gómez-García de la Banda M; Pediatric Neurology and ICU Department, AP-HP Université Paris Saclay, DMU Santé de l'Enfant et de l'Adolescent, Hôpital Raymond Poincaré, Garches, France.; Centre de Référence des Maladies Neuromusculaires Garches-Necker-Mondor-Hendaye (GNMH), Centre Nord- Est- Ile de France, Réseau National des Maladies Neuromusculaires, FILNEMUS, France.; European Reference Center Network (Euro-NMD ERN), Paris, France.
Amaddeo A; Pediatric Noninvasive Ventilation and Sleep Unit, AP-HP Hôpital Necker Enfants-Malades, Paris, France.; Université de Paris, VIFASOM, Paris, France.
Khirani S; Pediatric Noninvasive Ventilation and Sleep Unit, AP-HP Hôpital Necker Enfants-Malades, Paris, France.; Université de Paris, VIFASOM, Paris, France.; ASV Santé, Gennevilliers, France.
Pruvost S; Pediatric Neurology and ICU Department, AP-HP Université Paris Saclay, DMU Santé de l'Enfant et de l'Adolescent, Hôpital Raymond Poincaré, Garches, France.; Centre de Référence des Maladies Neuromusculaires Garches-Necker-Mondor-Hendaye (GNMH), Centre Nord- Est- Ile de France, Réseau National des Maladies Neuromusculaires, FILNEMUS, France.; European Reference Center Network (Euro-NMD ERN), Paris, France.
Barnerias C; Centre de Référence des Maladies Neuromusculaires Garches-Necker-Mondor-Hendaye (GNMH), Centre Nord- Est- Ile de France, Réseau National des Maladies Neuromusculaires, FILNEMUS, France.; European Reference Center Network (Euro-NMD ERN), Paris, France.; Pediatric Neurology Department, AP-HP Hôpital Necker Enfants Malades, Paris, France.
Dabaj I; Pediatric Neurology and ICU Department, AP-HP Université Paris Saclay, DMU Santé de l'Enfant et de l'Adolescent, Hôpital Raymond Poincaré, Garches, France.; Centre de Référence des Maladies Neuromusculaires Garches-Necker-Mondor-Hendaye (GNMH), Centre Nord- Est- Ile de France, Réseau National des Maladies Neuromusculaires, FILNEMUS, France.; Service de Néonatologie, Réanimation Pédiatrique, Neuropédiatrie et éDucation Fonctionnelle de l'enfant, CHU de Rouen, INSERM U 1245, ED497, Rouen, France.
Bénézit A; Pediatric Neurology and ICU Department, AP-HP Université Paris Saclay, DMU Santé de l'Enfant et de l'Adolescent, Hôpital Raymond Poincaré, Garches, France.; Centre de Référence des Maladies Neuromusculaires Garches-Necker-Mondor-Hendaye (GNMH), Centre Nord- Est- Ile de France, Réseau National des Maladies Neuromusculaires, FILNEMUS, France.; European Reference Center Network (Euro-NMD ERN), Paris, France.
Durigneux J; Pediatric Neurology Department, Centre de Référence des Maladies Neuromusculaires Atlantique Occitanie Caraïbes (AOC), Centre Hospitalier Universitaire, Angers, France.
Carlier RY; Centre de Référence des Maladies Neuromusculaires Garches-Necker-Mondor-Hendaye (GNMH), Centre Nord- Est- Ile de France, Réseau National des Maladies Neuromusculaires, FILNEMUS, France.; European Reference Center Network (Euro-NMD ERN), Paris, France.; Radiology Department, AP-HP Université Paris Saclay, DMU Smart Imaging, Hôpital Raymond Poincaré, Garches, France.
Desguerre I; Pediatric Neurology and ICU Department, AP-HP Université Paris Saclay, DMU Santé de l'Enfant et de l'Adolescent, Hôpital Raymond Poincaré, Garches, France.; Centre de Référence des Maladies Neuromusculaires Garches-Necker-Mondor-Hendaye (GNMH), Centre Nord- Est- Ile de France, Réseau National des Maladies Neuromusculaires, FILNEMUS, France.; European Reference Center Network (Euro-NMD ERN), Paris, France.; Pediatric Neurology Department, AP-HP Hôpital Necker Enfants Malades, Paris, France.
Quijano-Roy S; Pediatric Neurology and ICU Department, AP-HP Université Paris Saclay, DMU Santé de l'Enfant et de l'Adolescent, Hôpital Raymond Poincaré, Garches, France.; Centre de Référence des Maladies Neuromusculaires Garches-Necker-Mondor-Hendaye (GNMH), Centre Nord- Est- Ile de France, Réseau National des Maladies Neuromusculaires, FILNEMUS, France.; European Reference Center Network (Euro-NMD ERN), Paris, France.; INSERM U1179, Université de Versailles Saint-Quentin (UVSQ), UFR des Sciences de la Santé Simone Vieil, Montigny, France.
Fauroux B; Pediatric Noninvasive Ventilation and Sleep Unit, AP-HP Hôpital Necker Enfants-Malades, Paris, France.; Université de Paris, VIFASOM, Paris, France.
Pokaż więcej
Źródło :
Pediatric pulmonology [Pediatr Pulmonol] 2021 Jan; Vol. 56 (1), pp. 299-306. Date of Electronic Publication: 2020 Nov 05.
Typ publikacji :
Journal Article
MeSH Terms :
Motor Skills/*drug effects
Oligonucleotides/*therapeutic use
Respiratory Muscles/*drug effects
Spinal Muscular Atrophies of Childhood/*drug therapy
Child ; Child, Preschool ; Female ; Historically Controlled Study ; Humans ; Male ; Oligonucleotides/pharmacology ; Respiratory Function Tests ; Respiratory Muscles/physiopathology ; Spinal Muscular Atrophies of Childhood/physiopathology
Czasopismo naukowe

Ta witryna wykorzystuje pliki cookies do przechowywania informacji na Twoim komputerze. Pliki cookies stosujemy w celu świadczenia usług na najwyższym poziomie, w tym w sposób dostosowany do indywidualnych potrzeb. Korzystanie z witryny bez zmiany ustawień dotyczących cookies oznacza, że będą one zamieszczane w Twoim komputerze. W każdym momencie możesz dokonać zmiany ustawień dotyczących cookies