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Wyszukujesz frazę ""MENDELL, JERRY R."" wg kryterium: Autor


Tytuł :
Open-Label Evaluation of Eteplirsen in Patients with Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping: PROMOVI Trial.
Autorzy :
McDonald CM; Departments of Physical Medicine & Rehabilitation and Pediatrics, University of California Davis Health System and School of Medicine, Sacramento, CA, USA.
Shieh PB; Department of Neurology, University of California, Los Angeles, Los Angeles, CA, USA.
Abdel-Hamid HZ; UPMC Children's Hospital of Pittsburgh, Pittsburgh, PA, USA.
Connolly AM; Pediatrics, Center for Gene Therapy, Nationwide Children's Hospital, Columbus, OH, USA.
Ciafaloni E; University of Rochester Medical Center, Rochester, NY, USA.
Wagner KR; Center for Genetic Muscle Disorders, Kennedy Krieger Institute, and Departments of Neurology and Neuroscience, Johns Hopkins School of Medicine, Baltimore, MD, USA.
Goemans N; Department of Pediatrics and Child Neurology, University Hospitals Leuven, Leuven, Belgium.
Mercuri E; Pediatric Neurology, Catholic University, Rome, Italy.
Khan N; Sarepta Therapeutics, Inc., Cambridge, MA, USA.
Koenig E; Sarepta Therapeutics, Inc., Cambridge, MA, USA.
Malhotra J; Sarepta Therapeutics, Inc., Cambridge, MA, USA.
Zhang W; Sarepta Therapeutics, Inc., Cambridge, MA, USA.
Han B; Sarepta Therapeutics, Inc., Cambridge, MA, USA.
Mendell JR; Pediatrics, Center for Gene Therapy, Nationwide Children's Hospital, Columbus, OH, USA.
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Corporate Authors :
the Italian DMD Telethon Registry Study Group, Leuven NMRC Registry Investigators, CINRG Duchenne Natural History Investigators, and PROMOVI Trial Clinical Investigators
Źródło :
Journal of neuromuscular diseases [J Neuromuscul Dis] 2021 Jun 08. Date of Electronic Publication: 2021 Jun 08.
Typ publikacji :
Journal Article
Czasopismo naukowe
Tytuł :
Five-Year Extension Results of the Phase 1 START Trial of Onasemnogene Abeparvovec in Spinal Muscular Atrophy.
Autorzy :
Mendell JR; Center for Gene Therapy, Nationwide Children's Hospital, Columbus, Ohio.; Department of Pediatrics, The Ohio State University, Columbus.; Department of Neurology, The Ohio State University, Columbus.
Al-Zaidy SA; Al-Zaidy and Associates, LLC, Columbus, Ohio.
Lehman KJ; Center for Gene Therapy, Nationwide Children's Hospital, Columbus, Ohio.
McColly M; Center for Gene Therapy, Nationwide Children's Hospital, Columbus, Ohio.
Lowes LP; Center for Gene Therapy, Nationwide Children's Hospital, Columbus, Ohio.; Department of Pediatrics, The Ohio State University, Columbus.
Alfano LN; Center for Gene Therapy, Nationwide Children's Hospital, Columbus, Ohio.
Reash NF; Center for Gene Therapy, Nationwide Children's Hospital, Columbus, Ohio.
Iammarino MA; Center for Gene Therapy, Nationwide Children's Hospital, Columbus, Ohio.
Church KR; Center for Gene Therapy, Nationwide Children's Hospital, Columbus, Ohio.
Kleyn A; Novartis Gene Therapies, Inc, Bannockburn, Illinois.
Meriggioli MN; Novartis Gene Therapies, Inc, Bannockburn, Illinois.
Shell R; Department of Pediatrics, The Ohio State University, Columbus.; Section of Pulmonary Medicine, Department of Pediatrics, Nationwide Children's Hospital, Columbus, Ohio.
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Źródło :
JAMA neurology [JAMA Neurol] 2021 May 17. Date of Electronic Publication: 2021 May 17.
Typ publikacji :
Journal Article
Czasopismo naukowe
Tytuł :
Assessing Dysferlinopathy Patients Over Three Years With a New Motor Scale.
Autorzy :
Jacobs MB; Center for Translational Science, Division of Biostatistics and Study Methodology, Children's National Health System, Washington, DC.; Pediatrics, Epidemiology, and Biostatistics, George Washington University, Washington, DC.
James MK; The John Walton Muscular Dystrophy Research Centre, Translational and Clinical Research Institute, Newcastle University and Newcastle Hospitals NHS Foundation Trust, Central Parkway, Newcastle upon Tyne, UK.
Lowes LP; The Abigail Wexner Research Institute at Nationwide Children's Hospital, Columbus, OH.
Alfano LN; The Abigail Wexner Research Institute at Nationwide Children's Hospital, Columbus, OH.
Eagle M; The John Walton Muscular Dystrophy Research Centre, Translational and Clinical Research Institute, Newcastle University and Newcastle Hospitals NHS Foundation Trust, Central Parkway, Newcastle upon Tyne, UK.
Muni Lofra R; The John Walton Muscular Dystrophy Research Centre, Translational and Clinical Research Institute, Newcastle University and Newcastle Hospitals NHS Foundation Trust, Central Parkway, Newcastle upon Tyne, UK.
Moore U; The John Walton Muscular Dystrophy Research Centre, Translational and Clinical Research Institute, Newcastle University and Newcastle Hospitals NHS Foundation Trust, Central Parkway, Newcastle upon Tyne, UK.
Feng J; Center for Translational Science, Division of Biostatistics and Study Methodology, Children's National Health System, Washington, DC.
Rufibach LE; The Jain Foundation, Seattle, WA.
Rose K; The Children's Hospital at Westmead, The University of Sydney, Sydney, Australia.
Duong T; Cooperative International Neuromuscular Research Group (CINRG), Children's National Health System, Washington, DC.; Lucile Salter Packard Children's Hospital at Stanford, Palo Alto, CA.
Bello L; Department of Neuroscience, University of Padova, Padova, Italy.
Pedrosa-Hernández I; Physical Medicine and Rehabilitation, Hospital de la Santa Creu i Sant Pau, Barcelona, Spain.
Holsten S; Neuroscience Institute, Carolinas Neuromuscular/ALS-MDA Center, Carolinas HealthCare System, Charlotte, NC.
Sakamoto C; Department of Physical Rehabilitation, National Center Hospital, National Center of Neurology and Psychiatry, Tokyo, Japan.
Canal A; Institut de Myologie, AP-HP, GH Pitié-Salpêtrière, Paris, France.
Sanchez-Aguilera Práxedes N; Neurorehabilitation Unit, Rehabilitation Hospital Universitario Virgen del Rocío, Sevilla, Spain.
Thiele S; Friedrich-Baur-Institute, Department of Neurology, Ludwig-Maximilians University of Munich, Munich, Germany.
Siener C; Department of Neurology Washington University School of Medicine, St. Louis, MO.
Vandevelde B; Service des Maladies Neuromusculaire et de la SLA, Hôpital de La Timone, Marseille, France.
DeWolf B; Cooperative International Neuromuscular Research Group (CINRG), Children's National Health System, Washington, DC.
Maron E; ELAN-PHYSIO, Praxis für Physiotherapie Maron, Berlin, Germany.
Guglieri M; The John Walton Muscular Dystrophy Research Centre, Translational and Clinical Research Institute, Newcastle University and Newcastle Hospitals NHS Foundation Trust, Central Parkway, Newcastle upon Tyne, UK.
Hogrel JY; Institut de Myologie, AP-HP, GH Pitié-Salpêtrière, Paris, France.
Blamire AM; Magnetic Resonance Centre, Institute for Cellular Medicine, Newcastle University, Newcastle upon Tyne, UK.
Carlier PG; AIM & CEA NMR Laboratory, Institute of Myology, Pitié-Salpêtrière University Hospital, Paris, France.
Spuler S; Charite Muscle Research Unit, Experimental and Clinical Research Center, a joint cooperation of the Charité Medical Faculty and the Max Delbrück Center for Molecular Medicine, Berlin, Germany.
Day JW; Department of Neurology and Neurological Sciences, Stanford University School of Medicine, Stanford, CA.
Jones KJ; The Children's Hospital at Westmead, The University of Sydney, Sydney, Australia.
Bharucha-Goebel DX; Department of Neurology Children's National Health System, Washington, DC.; National Institutes of Health (NINDS), Bethesda, MD.
Salort-Campana E; Service des Maladies Neuromusculaire et de la SLA, Hôpital de La Timone, Marseille, France.
Pestronk A; Department of Neurology Washington University School of Medicine, St. Louis, MO.
Walter MC; Friedrich-Baur-Institute, Department of Neurology, Ludwig-Maximilians University of Munich, Munich, Germany.
Paradas C; Neuromuscular Unit, Department of Neurology, Hospital U. Virgen del Rocío/Instituto de Biomedicina de Sevilla, Sevilla, Spain.
Stojkovic T; Institut de Myologie, AP-HP, GH Pitié-Salpêtrière, Paris, France.
Mori-Yoshimura M; Department of Neurology, National Center Hospital, National Center of Neurology and Psychiatry, Tokyo, Japan.
Bravver E; Neuroscience Institute, Carolinas Neuromuscular/ALS-MDA Center, Carolinas HealthCare System, Charlotte, NC.
Díaz-Manera J; Centro de Investigación Biomédica en Red en Enfermedades Raras (CIBERER), Barcelona, Spain.; Neuromuscular Disorders Unit, Neurology Department, Hospital de la Santa Creu i Sant Pau, Barcelona, Spain.
Pegoraro E; Department of Neuroscience, University of Padova, Padova, Italy.
Mendell JR; The Abigail Wexner Research Institute at Nationwide Children's Hospital, Columbus, OH.
Mayhew AG; The John Walton Muscular Dystrophy Research Centre, Translational and Clinical Research Institute, Newcastle University and Newcastle Hospitals NHS Foundation Trust, Central Parkway, Newcastle upon Tyne, UK.
Straub V; The John Walton Muscular Dystrophy Research Centre, Translational and Clinical Research Institute, Newcastle University and Newcastle Hospitals NHS Foundation Trust, Central Parkway, Newcastle upon Tyne, UK.
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Corporate Authors :
Jain COS Consortium
Źródło :
Annals of neurology [Ann Neurol] 2021 May; Vol. 89 (5), pp. 967-978. Date of Electronic Publication: 2021 Feb 26.
Typ publikacji :
Journal Article; Research Support, Non-U.S. Gov't
Czasopismo naukowe
Tytuł :
Spinal Muscular Atrophy.
Autorzy :
Nicolau S; Center for Gene Therapy, The Abigail Wexner Research Institute, Nationwide Children's Hospital, Columbus, OH. Electronic address: .
Waldrop MA; Center for Gene Therapy, The Abigail Wexner Research Institute, Nationwide Children's Hospital, Columbus, OH; Departments of Pediatrics and Neurology, Ohio State University, Columbus, OH.
Connolly AM; Center for Gene Therapy, The Abigail Wexner Research Institute, Nationwide Children's Hospital, Columbus, OH; Departments of Pediatrics and Neurology, Ohio State University, Columbus, OH.
Mendell JR; Center for Gene Therapy, The Abigail Wexner Research Institute, Nationwide Children's Hospital, Columbus, OH; Departments of Pediatrics and Neurology, Ohio State University, Columbus, OH.
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Źródło :
Seminars in pediatric neurology [Semin Pediatr Neurol] 2021 Apr; Vol. 37, pp. 100878. Date of Electronic Publication: 2021 Feb 11.
Typ publikacji :
Journal Article; Review
Czasopismo naukowe
Tytuł :
Therapeutic Approaches for Duchenne Muscular Dystrophy: Old and New.
Autorzy :
Mackenzie SJ; Center for Gene Therapy, Nationwide Children's Hospital, Columbus, OH; Department of Pediatrics and Neurology; The Ohio State University, Columbus, OH. Electronic address: .
Nicolau S; Center for Gene Therapy, Nationwide Children's Hospital, Columbus, OH.
Connolly AM; Center for Gene Therapy, Nationwide Children's Hospital, Columbus, OH; Department of Pediatrics and Neurology; The Ohio State University, Columbus, OH.
Mendell JR; Center for Gene Therapy, Nationwide Children's Hospital, Columbus, OH; Department of Pediatrics and Neurology; The Ohio State University, Columbus, OH.
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Źródło :
Seminars in pediatric neurology [Semin Pediatr Neurol] 2021 Apr; Vol. 37, pp. 100877. Date of Electronic Publication: 2021 Feb 11.
Typ publikacji :
Journal Article; Review
Czasopismo naukowe
Tytuł :
Miyoshi myopathy and limb girdle muscular dystrophy R2 are the same disease.
Autorzy :
Moore U; The John Walton Muscular Dystrophy Research Centre, Translational and Clinical Research Institute, Newcastle University and Newcastle Hospitals NHS Foundation Trust, Central Parkway, Newcastle upon Tyne, United Kingdom.
Gordish H; Center for Translational Science, Division of Biostatistics and Study Methodology, Children's National Health System, Washington, DC, United States; Pediatrics, Epidemiology and Biostatistics, George Washington University, Washington, DC, United States.
Diaz-Manera J; Centro de Investigación Biomédica en Red en Enfermedades Raras (CIBERER), Barcelona, Spain; Neuromuscular Disorders Unit, Neurology Department, Hospital de la Santa Creu i Sant Pau.
James MK; The John Walton Muscular Dystrophy Research Centre, Translational and Clinical Research Institute, Newcastle University and Newcastle Hospitals NHS Foundation Trust, Central Parkway, Newcastle upon Tyne, United Kingdom.
Mayhew AG; The John Walton Muscular Dystrophy Research Centre, Translational and Clinical Research Institute, Newcastle University and Newcastle Hospitals NHS Foundation Trust, Central Parkway, Newcastle upon Tyne, United Kingdom.
Guglieri M; The John Walton Muscular Dystrophy Research Centre, Translational and Clinical Research Institute, Newcastle University and Newcastle Hospitals NHS Foundation Trust, Central Parkway, Newcastle upon Tyne, United Kingdom.
Fernandez-Torron R; The John Walton Muscular Dystrophy Research Centre, Translational and Clinical Research Institute, Newcastle University and Newcastle Hospitals NHS Foundation Trust, Central Parkway, Newcastle upon Tyne, United Kingdom.
Rufibach LE; The Jain Foundation, Seattle, Washington DC, United States.
Feng J; Center for Translational Science, Division of Biostatistics and Study Methodology, Children's National Health System, Washington, DC, United States.
Blamire AM; Magnetic Resonance Centre, Translational and Clinical Research Institute, Newcastle University, United Kingdom.
Carlier PG; AIM & CEA NMR Laboratory, Institute of Myology, Pitié-Salpêtrière University Hospital, 47-83 Paris, France.
Spuler S; Charite Muscle Research Unit, Experimental and Clinical Research Center, a Joint Cooperation of the Charité Medical Faculty and the Max Delbrück Center for Molecular Medicine, Berlin, Germany.
Day JW; Department of Neurology and Neurological Sciences, Stanford University School of Medicine, Stanford, CA, United States.
Jones KJ; The Children's Hospital at Westmead, and The University of Sydney, Australia.
Bharucha-Goebel DX; Department of Neurology Children's National Health System, Washington, DC, United States; National Institutes of Health (NINDS), Bethesda, MD, United States.
Salort-Campana E; Service des maladies neuromusculaire et de la SLA, Hôpital de La Timone, Marseille, France.
Pestronk A; Department of Neurology Washington University School of Medicine, St. Louis, MO, United States.
Walter MC; Friedrich-Baur-Institute, Department of Neurology, Ludwig-Maximilians-University of Munich, Germany.
Paradas C; Neuromuscular Unit, Department of Neurology, Hospital U. Virgen del Rocío/Instituto de Biomedicina de Sevilla, Sevilla, Spain.
Stojkovic T; Centre de référence des maladies neuromusculaires, Institut de Myologie, AP-HP, Sorbonne Université, Hôpital Pitié-Salpêtrière, Paris, France.
Mori-Yoshimura M; Department of Neurology, National Center Hospital, National Center of Neurology and Psychiatry Tokyo, Japan.
Bravver E; Neuroscience Institute, Carolinas Neuromuscular/ALS-MDA Center, Carolinas HealthCare System, Charlotte, NC, United States.
Pegoraro E; Department of Neuroscience, University of Padova, Italy.
Lowes LP; The Abigail Wexner Research Institute at Nationwide Children's Hospital, Columbus, OH United States.
Mendell JR; The Abigail Wexner Research Institute at Nationwide Children's Hospital, Columbus, OH United States.
Bushby K; The John Walton Muscular Dystrophy Research Centre, Translational and Clinical Research Institute, Newcastle University and Newcastle Hospitals NHS Foundation Trust, Central Parkway, Newcastle upon Tyne, United Kingdom.
Straub V; The John Walton Muscular Dystrophy Research Centre, Translational and Clinical Research Institute, Newcastle University and Newcastle Hospitals NHS Foundation Trust, Central Parkway, Newcastle upon Tyne, United Kingdom. Electronic address: .
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Corporate Authors :
Jain COS Consortium
Źródło :
Neuromuscular disorders : NMD [Neuromuscul Disord] 2021 Apr; Vol. 31 (4), pp. 265-280. Date of Electronic Publication: 2021 Jan 21.
Typ publikacji :
Journal Article
Czasopismo naukowe
Tytuł :
Onasemnogene abeparvovec gene therapy for symptomatic infantile-onset spinal muscular atrophy in patients with two copies of SMN2 (STR1VE): an open-label, single-arm, multicentre, phase 3 trial.
Autorzy :
Day JW; Department of Neurology, Stanford University Medical Center, Palo Alto, CA, USA. Electronic address: .
Finkel RS; Department of Pediatrics, Nemours Children's Hospital, Orlando, FL, USA; Center for Experimental Neurotherapeutics, St Jude Children's Research Hospital, Memphis, TN, USA.
Chiriboga CA; Division of Pediatric Neurology, Columbia University Medical Center, New York, NY, USA.
Connolly AM; Department of Neurology, Nationwide Children's Hospital, Columbus, OH, USA; Center for Gene Therapy, Nationwide Children's Hospital, Columbus, OH, USA; Department of Pediatrics, Ohio State University, Columbus, OH, USA; Department of Neurology, Ohio State University, Columbus, OH, USA.
Crawford TO; Department of Neurology, Johns Hopkins Medicine, Baltimore, MD, USA; Department of Pediatrics, Johns Hopkins Medicine, Baltimore, MD, USA.
Darras BT; Department of Neurology, Boston Children's Hospital, Boston, MA, USA.
Iannaccone ST; Department of Pediatrics, University of Texas Southwestern Medical Center, Dallas, TX, USA.
Kuntz NL; Division of Neurology, Ann and Robert H Lurie Children's Hospital of Chicago, Chicago, IL, USA.
Peña LDM; Division of Human Genetics, Cincinnati Children's Hospital, Cincinnati, OH, USA; Department of Pediatrics, University of Cincinnati College of Medicine, Cincinnati OH, USA.
Shieh PB; Department of Neurology, David Geffen School of Medicine at UCLA, Los Angeles, CA, USA.
Smith EC; Department of Pediatrics, Duke University School of Medicine, Durham, NC, USA.
Kwon JM; Department of Neurology, University of Wisconsin School of Medicine and Public Health, Madison, WI, USA.
Zaidman CM; Department of Neurology, Washington University School of Medicine, St Louis, MO, USA.
Schultz M; Novartis Gene Therapies, Bannockburn, IL, USA.
Feltner DE; Novartis Gene Therapies, Bannockburn, IL, USA.
Tauscher-Wisniewski S; Novartis Gene Therapies, Bannockburn, IL, USA.
Ouyang H; Novartis Gene Therapies, Bannockburn, IL, USA.
Chand DH; Department of Pediatrics, Washington University School of Medicine, St Louis, MO, USA; Novartis Gene Therapies, Bannockburn, IL, USA.
Sproule DM; Novartis Gene Therapies, Bannockburn, IL, USA.
Macek TA; Novartis Gene Therapies, Bannockburn, IL, USA.
Mendell JR; Center for Gene Therapy, Nationwide Children's Hospital, Columbus, OH, USA; Department of Pediatrics, Ohio State University, Columbus, OH, USA; Department of Neurology, Ohio State University, Columbus, OH, USA.
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Źródło :
The Lancet. Neurology [Lancet Neurol] 2021 Apr; Vol. 20 (4), pp. 284-293. Date of Electronic Publication: 2021 Mar 17.
Typ publikacji :
Clinical Trial, Phase III; Journal Article; Multicenter Study; Research Support, Non-U.S. Gov't
MeSH Terms :
Biological Products/*therapeutic use
Genetic Therapy/*methods
Recombinant Fusion Proteins/*therapeutic use
Spinal Muscular Atrophies of Childhood/*drug therapy
Spinal Muscular Atrophies of Childhood/*genetics
Child, Preschool ; Female ; Humans ; Infant ; Male ; Survival of Motor Neuron 2 Protein/genetics ; Treatment Outcome
Czasopismo naukowe
Tytuł :
Dose-Escalation Study of Systemically Delivered rAAVrh74.MHCK7.micro-dystrophin in the mdx Mouse Model of Duchenne Muscular Dystrophy.
Autorzy :
Potter RA; Sarepta Therapeutics, Inc., Cambridge, Massachusetts, USA.; Center for Gene Therapy, The Research Institute at Nationwide Children's Hospital, Columbus, Ohio, USA.
Griffin DA; Sarepta Therapeutics, Inc., Cambridge, Massachusetts, USA.; Center for Gene Therapy, The Research Institute at Nationwide Children's Hospital, Columbus, Ohio, USA.
Heller KN; Center for Gene Therapy, The Research Institute at Nationwide Children's Hospital, Columbus, Ohio, USA.
Peterson EL; Sarepta Therapeutics, Inc., Cambridge, Massachusetts, USA.; Center for Gene Therapy, The Research Institute at Nationwide Children's Hospital, Columbus, Ohio, USA.
Clark EK; Center for Gene Therapy, The Research Institute at Nationwide Children's Hospital, Columbus, Ohio, USA.
Mendell JR; Center for Gene Therapy, The Research Institute at Nationwide Children's Hospital, Columbus, Ohio, USA.; Department of Pediatrics and Neurology, The Ohio State University, Columbus, Ohio, USA.
Rodino-Klapac LR; Sarepta Therapeutics, Inc., Cambridge, Massachusetts, USA.; Center for Gene Therapy, The Research Institute at Nationwide Children's Hospital, Columbus, Ohio, USA.; Department of Pediatrics and Neurology, The Ohio State University, Columbus, Ohio, USA.
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Źródło :
Human gene therapy [Hum Gene Ther] 2021 Apr; Vol. 32 (7-8), pp. 375-389. Date of Electronic Publication: 2021 Feb 18.
Typ publikacji :
Journal Article
Czasopismo naukowe
Tytuł :
Health-Related Quality of Life and Emotional Distress Among Mothers of Sons With Muscular Dystrophy as Compared to Sex- and Age Group-Matched Controls.
Autorzy :
Jackson JL; Center for Biobehavioral Health, The Abigail Wexner Research Institute at 2650Nationwide Children's Hospital, OH, USA.; Department of Pediatrics, 12305The Ohio State University, OH, USA.
Korth CX; Center for Biobehavioral Health, The Abigail Wexner Research Institute at 2650Nationwide Children's Hospital, OH, USA.
Leslie CE; Center for Biobehavioral Health, The Abigail Wexner Research Institute at 2650Nationwide Children's Hospital, OH, USA.
Cotto J; Center for Biobehavioral Health, The Abigail Wexner Research Institute at 2650Nationwide Children's Hospital, OH, USA.
Mah ML; The Heart Center, 2650Nationwide Children's Hospital, OH, USA.
Hor K; The Heart Center, 2650Nationwide Children's Hospital, OH, USA.
Cripe L; The Heart Center, 2650Nationwide Children's Hospital, OH, USA.
Al-Zaidy S; Department of Pediatrics, 12305The Ohio State University, OH, USA.
Camino EM; Center for Gene Therapy, The Abigail Wexner Research Institute at 2650Nationwide Children's Hospital, OH, USA.
Church K; Center for Gene Therapy, The Abigail Wexner Research Institute at 2650Nationwide Children's Hospital, OH, USA.
Lehman KJ; Center for Gene Therapy, The Abigail Wexner Research Institute at 2650Nationwide Children's Hospital, OH, USA.
Shay V; The Heart Center, 2650Nationwide Children's Hospital, OH, USA.
Mendell JR; Department of Pediatrics, 12305The Ohio State University, OH, USA.; Center for Gene Therapy, The Abigail Wexner Research Institute at 2650Nationwide Children's Hospital, OH, USA.
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Źródło :
Journal of child neurology [J Child Neurol] 2021 Mar; Vol. 36 (3), pp. 177-185. Date of Electronic Publication: 2020 Oct 09.
Typ publikacji :
Journal Article
Czasopismo naukowe
Tytuł :
Adeno-associated virus serotype 9 antibodies in patients screened for treatment with onasemnogene abeparvovec.
Autorzy :
Day JW; Division of Neuromuscular Medicine, Stanford Neuroscience Health Center, MC 5979, 213 Quarry Road, Palo Alto, CA 94304, USA.
Finkel RS; Center for Experimental Neurotherapeutics, St. Jude Children's Research Hospital, 262 Danny Thomas Place, Memphis, TN 38105, USA.; Department of Pediatrics, Nemours Children's Hospital, Orlando, FL, USA.
Mercuri E; Department of Paediatric Neurology and Nemo Clinical Centre, Catholic University, Largo Agostino Gemelli 8, 00168 Rome, Italy.
Swoboda KJ; Department of Neurology, Massachusetts General Hospital for Children, 185 Cambridge Street no. 5-240, Boston, MA 02114, USA.
Menier M; Novartis Gene Therapies, Inc., 2275 Half Day Road, Suite 200, Bannockburn, IL 60015, USA.
van Olden R; Novartis Gene Therapies, Inc., 2275 Half Day Road, Suite 200, Bannockburn, IL 60015, USA.
Tauscher-Wisniewski S; Novartis Gene Therapies, Inc., 2275 Half Day Road, Suite 200, Bannockburn, IL 60015, USA.
Mendell JR; Center for Gene Therapy, Nationwide Children's Hospital, 700 Children's Drive, Columbus, OH 43205, USA.; Departments of Pediatrics and Neurology, The Ohio State University, 410 West 10th Avenue, Columbus, OH 43210, USA.
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Źródło :
Molecular therapy. Methods & clinical development [Mol Ther Methods Clin Dev] 2021 Feb 24; Vol. 21, pp. 76-82. Date of Electronic Publication: 2021 Feb 24 (Print Publication: 2021).
Typ publikacji :
Journal Article
Czasopismo naukowe
Tytuł :
Comparison of Long-term Ambulatory Function in Patients with Duchenne Muscular Dystrophy Treated with Eteplirsen and Matched Natural History Controls.
Autorzy :
Mendell JR; Center for Gene Therapy, The Research Institute at Nationwide Children's Hospital, Columbus, OH, USA.; Department of Pediatrics and Neurology, The Ohio State University, Columbus, OH, USA.
Khan N; Sarepta Therapeutics, Inc., Cambridge, MA, USA.
Sha N; Sarepta Therapeutics, Inc., Cambridge, MA, USA.
Eliopoulos H; Sarepta Therapeutics, Inc., Cambridge, MA, USA.
McDonald CM; University of California Davis Medical Center, Sacramento, CA, USA.
Goemans N; University Hospitals Leuven, Leuven, Belgium.
Mercuri E; Paediatric Neurology and Centro Clinico Nemo, Catholic University and Policlinico Gemelli.
Lowes LP; Center for Gene Therapy, The Research Institute at Nationwide Children's Hospital, Columbus, OH, USA.; Department of Pediatrics and Neurology, The Ohio State University, Columbus, OH, USA.
Alfano LN; Center for Gene Therapy, The Research Institute at Nationwide Children's Hospital, Columbus, OH, USA.
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Corporate Authors :
Eteplirsen Study Group
Źródło :
Journal of neuromuscular diseases [J Neuromuscul Dis] 2021 Feb 19. Date of Electronic Publication: 2021 Feb 19.
Typ publikacji :
Journal Article
Czasopismo naukowe
Tytuł :
AAV1.NT-3 gene therapy for X-linked Charcot-Marie-Tooth neuropathy type 1.
Autorzy :
Ozes B; Center for Gene Therapy, The Abigail Wexner Research Institute at Nationwide Children's Hospital, Columbus, OH, USA.
Myers M; Center for Gene Therapy, The Abigail Wexner Research Institute at Nationwide Children's Hospital, Columbus, OH, USA.
Moss K; Center for Gene Therapy, The Abigail Wexner Research Institute at Nationwide Children's Hospital, Columbus, OH, USA.
Mckinney J; Department of Pediatrics and Neurology, Nationwide Children's Hospital and The Ohio State University, Columbus, OH, USA.
Ridgley A; Center for Gene Therapy, The Abigail Wexner Research Institute at Nationwide Children's Hospital, Columbus, OH, USA.
Chen L; Center for Gene Therapy, The Abigail Wexner Research Institute at Nationwide Children's Hospital, Columbus, OH, USA.
Bai S; Department of Biomedical Informatics, The Ohio State University College of Medicine, Columbus, OH, USA.; Biostatistics Resource at Nationwide Children's Hospital, Columbus, OH, USA.
Abrams CK; Department of Neurology and Rehabilitation, University of Illinois at Chicago, Chicago, IL, USA.
Freidin MM; Department of Neurology and Rehabilitation, University of Illinois at Chicago, Chicago, IL, USA.
Mendell JR; Center for Gene Therapy, The Abigail Wexner Research Institute at Nationwide Children's Hospital, Columbus, OH, USA.; Department of Pediatrics and Neurology, Nationwide Children's Hospital and The Ohio State University, Columbus, OH, USA.
Sahenk Z; Center for Gene Therapy, The Abigail Wexner Research Institute at Nationwide Children's Hospital, Columbus, OH, USA. .; Department of Pediatrics and Neurology, Nationwide Children's Hospital and The Ohio State University, Columbus, OH, USA. .; Department of Pathology and Laboratory Medicine, Nationwide Children's Hospital, Columbus, OH, USA. .
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Źródło :
Gene therapy [Gene Ther] 2021 Feb 04. Date of Electronic Publication: 2021 Feb 04.
Typ publikacji :
Journal Article
Czasopismo naukowe
Tytuł :
Current Clinical Applications of In Vivo Gene Therapy with AAVs.
Autorzy :
Mendell JR; Center of Gene Therapy, Abigail Wexner Research Institute, Nationwide Children's Hospital, Columbus, OH, USA; Department of Pediatrics and Neurology, The Ohio State University, Columbus, OH, USA.
Al-Zaidy SA; Al-Zaidy and Associates, LLC, Columbus, OH, USA.
Rodino-Klapac LR; Sarepta Therapeutics, Inc., Cambridge, MA, USA.
Goodspeed K; Department of Pediatrics, UT Southwestern Medical Center, Dallas, TX, USA.
Gray SJ; Department of Pediatrics, UT Southwestern Medical Center, Dallas, TX, USA.
Kay CN; Vitreoretinal Associates, Gainesville, FL, USA.
Boye SL; Department of Pediatrics, Powell Gene Therapy Center, University of Florida, Gainesville, FL, USA.
Boye SE; Division of Cellular and Molecular Therapeutics, University of Florida, Gainesville, FL, USA.
George LA; Division of Hematology and the Perelman Center for Cellular and Molecular Therapeutics, Philadelphia, PA, USA; Children's Hospital of Philadelphia, Philadelphia, PA, USA.
Salabarria S; Department of Pediatrics, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, PA, USA.
Corti M; Department of Pediatrics, College of Medicine, University of Florida, Gainesville, FL, USA; Powell Gene Therapy Center, University of Florida, Gainesville, FL, USA.
Byrne BJ; Department of Pediatrics, College of Medicine, University of Florida, Gainesville, FL, USA; Powell Gene Therapy Center, University of Florida, Gainesville, FL, USA.
Tremblay JP; Centre de Recherche du CHUQ-Université Laval, Québec, QC, Canada. Electronic address: .
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Źródło :
Molecular therapy : the journal of the American Society of Gene Therapy [Mol Ther] 2021 Feb 03; Vol. 29 (2), pp. 464-488. Date of Electronic Publication: 2020 Dec 10.
Typ publikacji :
Journal Article; Review; Research Support, N.I.H., Extramural; Research Support, Non-U.S. Gov't
Czasopismo naukowe
Tytuł :
Assessment of rAAVrh.74.MHCK7.micro-dystrophin Gene Therapy Using Magnetic Resonance Imaging in Children With Duchenne Muscular Dystrophy.
Autorzy :
Willcocks RJ; Department of Physical Therapy, University of Florida, Gainesville.
Forbes SC; Department of Physical Therapy, University of Florida, Gainesville.
Walter GA; Department of Physical Therapy, University of Florida, Gainesville.
Sweeney L; Department of Physical Therapy, University of Florida, Gainesville.
Rodino-Klapac LR; Sarepta Therapeutics Inc, Cambridge, Massachusetts.
Mendell JR; Center for Gene Therapy, The Research Institute at Nationwide Children's Hospital, Columbus, Ohio.
Vandenborne K; Department of Physical Therapy, University of Florida, Gainesville.
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Źródło :
JAMA network open [JAMA Netw Open] 2021 Jan 04; Vol. 4 (1), pp. e2031851. Date of Electronic Publication: 2021 Jan 04.
Typ publikacji :
Journal Article; Research Support, N.I.H., Extramural; Research Support, Non-U.S. Gov't; Research Support, U.S. Gov't, Non-P.H.S.
MeSH Terms :
Dystrophin/*genetics
Genetic Therapy/*methods
Magnetic Resonance Imaging/*methods
Muscular Dystrophy, Duchenne/*diagnostic imaging
Muscular Dystrophy, Duchenne/*therapy
Case-Control Studies ; Child ; Child, Preschool ; Humans ; Muscle, Skeletal/diagnostic imaging ; Treatment Outcome
Czasopismo naukowe
Tytuł :
Intensive Teenage Activity Is Associated With Greater Muscle Hyperintensity on T1W Magnetic Resonance Imaging in Adults With Dysferlinopathy.
Autorzy :
Moore U; The John Walton Muscular Dystrophy Research Centre, Translational and Clinical Research Institute, Newcastle University, Newcastle Hospitals NHS Foundation Trust, Newcastle upon Tyne, United Kingdom.
Jacobs M; Division of Biostatistics and Study Methodology, Center for Translational Science, Children's National Health System, Washington, DC, United States.; Pediatrics, Epidemiology and Biostatistics, George Washington University, Washington, DC, United States.
Fernandez-Torron R; The John Walton Muscular Dystrophy Research Centre, Translational and Clinical Research Institute, Newcastle University, Newcastle Hospitals NHS Foundation Trust, Newcastle upon Tyne, United Kingdom.; Neuromuscular Area, Biodonostia Health Research Institute, Neurology Service, Donostia University Hospital, Donostia-San Sebastian, Spain.
LLauger Rossello J; Radiology Department, Hospital de la Santa Creu i Sant Pau, Universitat Autònoma de Barcelona, Barcelona, Spain.
Smith FE; Magnetic Resonance Centre, Translational and Clinical Research Institute, Newcastle University, Newcastle upon Tyne, United Kingdom.
James M; The John Walton Muscular Dystrophy Research Centre, Translational and Clinical Research Institute, Newcastle University, Newcastle Hospitals NHS Foundation Trust, Newcastle upon Tyne, United Kingdom.
Mayhew A; The John Walton Muscular Dystrophy Research Centre, Translational and Clinical Research Institute, Newcastle University, Newcastle Hospitals NHS Foundation Trust, Newcastle upon Tyne, United Kingdom.
Rufibach L; Jain Foundation, Newcastle upon Tyne, Seattle, WA, United States.
Carlier PG; Université Paris-Saclay, CEA, DRF, Service Hospitalier Frederic Joliot, Orsay, France.
Blamire AM; Magnetic Resonance Centre, Translational and Clinical Research Institute, Newcastle University, Newcastle upon Tyne, United Kingdom.
Day JW; Department of Neurology and Neurological Sciences, Stanford University School of Medicine, Stanford, CA, United States.
Jones KJ; The Children's Hospital at Westmead, The University of Sydney, Sydney, NSW, Australia.
Bharucha-Goebel DX; Department of Neurology Children's National Health System, Washington, DC, United States.; National Institutes of Health (NINDS), Bethesda, MD, United States.
Salort-Campana E; Service des maladies neuromusculaire et de la SLA, Hôpital de La Timone, Marseille, France.
Pestronk A; Department of Neurology Washington University School of Medicine, St. Louis, MO, United States.
Walter MC; Department of Neurology, Friedrich-Baur-Institute, Ludwig-Maximilians-University of Munich, Munich, Germany.
Paradas C; Center for Biomedical Network Research on Eurodegenerative Diseases, Instituto de Salud Carlos III, Madrid, Spain.; Neuromuscular Unit, Department of Neurology, Hospital U. Virgen del Rocío/Instituto de Biomedicina de Sevilla, Sevilla, Spain.
Stojkovic T; Centre de référence des maladies neuromusculaires, Institut de Myologie, AP-HP, Sorbonne Université, Hôpital Pitié-Salpêtrière, Paris, France.
Mori-Yoshimura M; Department of Neurology, National Center Hospital, National Center of Neurology and Psychiatry Tokyo, Tokyo, Japan.
Bravver E; Carolinas Neuromuscular/ALS-MDA Center, Neuroscience Institute, Carolinas HealthCare System, Charlotte, NC, United States.
Pegoraro E; Department of Neuroscience, University of Padova, Padova, Italy.
Mendell JR; The Abigail Wexner Research Institute at Nationwide Children's Hospital, Columbus, OH, United States.
Bushby K; The John Walton Muscular Dystrophy Research Centre, Translational and Clinical Research Institute, Newcastle University, Newcastle Hospitals NHS Foundation Trust, Newcastle upon Tyne, United Kingdom.
Straub V; The John Walton Muscular Dystrophy Research Centre, Translational and Clinical Research Institute, Newcastle University, Newcastle Hospitals NHS Foundation Trust, Newcastle upon Tyne, United Kingdom.
Diaz-Manera J; The John Walton Muscular Dystrophy Research Centre, Translational and Clinical Research Institute, Newcastle University, Newcastle Hospitals NHS Foundation Trust, Newcastle upon Tyne, United Kingdom.; Neuromuscular disorders Unit, Department of Neurology, Hospital de la Santa Creu I Sant Pau, Barcelona, Spain.; Centro de Investigación Biomédica en Red en Enfermedades Raras (CIBERER), Valencia, Spain.
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Źródło :
Frontiers in neurology [Front Neurol] 2020 Dec 16; Vol. 11, pp. 613446. Date of Electronic Publication: 2020 Dec 16 (Print Publication: 2020).
Typ publikacji :
Journal Article
Czasopismo naukowe
Tytuł :
Natural History of Steroid-Treated Young Boys With Duchenne Muscular Dystrophy Using the NSAA, 100m, and Timed Functional Tests.
Autorzy :
Miller NF; Center for Gene Therapy, The Abigail Wexner Research Institute at Nationwide Children's Hospital, Columbus, Ohio. Electronic address: .
Alfano LN; Center for Gene Therapy, The Abigail Wexner Research Institute at Nationwide Children's Hospital, Columbus, Ohio.
Iammarino MA; Center for Gene Therapy, The Abigail Wexner Research Institute at Nationwide Children's Hospital, Columbus, Ohio.
Connolly AM; Center for Gene Therapy, The Abigail Wexner Research Institute at Nationwide Children's Hospital, Columbus, Ohio; Department of Neurology, The College of Medicine, The Ohio State University Wexner Medical Center, Columbus, Ohio.
Moore-Clingenpeel M; Biostatistics Resource, The Abigail Wexner Research Institute at Nationwide Children's Hospital, Columbus, Ohio.
Powers BR; Center for Gene Therapy, The Abigail Wexner Research Institute at Nationwide Children's Hospital, Columbus, Ohio.
Tsao CY; Department of Neurology, The College of Medicine, The Ohio State University Wexner Medical Center, Columbus, Ohio.
Waldrop MA; Center for Gene Therapy, The Abigail Wexner Research Institute at Nationwide Children's Hospital, Columbus, Ohio; Department of Pediatrics, The College of Medicine, The Ohio State University Wexner Medical Center, Columbus, Ohio.
Flanigan KM; Center for Gene Therapy, The Abigail Wexner Research Institute at Nationwide Children's Hospital, Columbus, Ohio; Department of Pediatrics, The College of Medicine, The Ohio State University Wexner Medical Center, Columbus, Ohio.
Mendell JR; Center for Gene Therapy, The Abigail Wexner Research Institute at Nationwide Children's Hospital, Columbus, Ohio; Department of Pediatrics, The College of Medicine, The Ohio State University Wexner Medical Center, Columbus, Ohio.
Lowes LP; Center for Gene Therapy, The Abigail Wexner Research Institute at Nationwide Children's Hospital, Columbus, Ohio; Department of Pediatrics, The College of Medicine, The Ohio State University Wexner Medical Center, Columbus, Ohio.
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Źródło :
Pediatric neurology [Pediatr Neurol] 2020 Dec; Vol. 113, pp. 15-20. Date of Electronic Publication: 2020 Aug 27.
Typ publikacji :
Journal Article
Czasopismo naukowe
Tytuł :
Duchenne and Becker muscular dystrophy carriers: Evidence of cardiomyopathy by exercise and cardiac MRI testing.
Autorzy :
Mah ML; The Heart Center, Nationwide Children's Hospital, Ohio State University, Columbus, OH, United States of America. Electronic address: .
Cripe L; The Heart Center, Nationwide Children's Hospital, Ohio State University, Columbus, OH, United States of America.
Slawinski MK; The Heart Center, Nationwide Children's Hospital, Ohio State University, Columbus, OH, United States of America.
Al-Zaidy SA; Center for Gene Therapy Research Institute at Nationwide Children's Hospital, Dept. of Pediatrics and Neurology, Ohio State University, Columbus, OH, United States of America.
Camino E; Center for Gene Therapy Research Institute at Nationwide Children's Hospital, Dept. of Pediatrics and Neurology, Ohio State University, Columbus, OH, United States of America.
Lehman KJ; Center for Gene Therapy Research Institute at Nationwide Children's Hospital, Dept. of Pediatrics and Neurology, Ohio State University, Columbus, OH, United States of America.
Jackson JL; Center for Biobehavioral Health, Nationwide Children's Hospital, Columbus, OH, United States of America.
Iammarino M; Center for Gene Therapy Research Institute at Nationwide Children's Hospital, Dept. of Pediatrics and Neurology, Ohio State University, Columbus, OH, United States of America.
Miller N; Center for Gene Therapy Research Institute at Nationwide Children's Hospital, Dept. of Pediatrics and Neurology, Ohio State University, Columbus, OH, United States of America.
Mendell JR; Center for Gene Therapy Research Institute at Nationwide Children's Hospital, Dept. of Pediatrics and Neurology, Ohio State University, Columbus, OH, United States of America.
Hor KN; The Heart Center, Nationwide Children's Hospital, Ohio State University, Columbus, OH, United States of America.
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Źródło :
International journal of cardiology [Int J Cardiol] 2020 Oct 01; Vol. 316, pp. 257-265. Date of Electronic Publication: 2020 May 27.
Typ publikacji :
Journal Article
MeSH Terms :
Cardiomyopathies*/diagnostic imaging
Cardiomyopathies*/epidemiology
Cardiomyopathies*/genetics
Muscular Dystrophy, Duchenne*/diagnostic imaging
Muscular Dystrophy, Duchenne*/epidemiology
Muscular Dystrophy, Duchenne*/genetics
Adult ; Contrast Media ; Gadolinium ; Humans ; Magnetic Resonance Imaging ; Magnetic Resonance Imaging, Cine ; Prospective Studies ; Stroke Volume ; Ventricular Function, Left
Czasopismo naukowe
Tytuł :
Assessment of Systemic Delivery of rAAVrh74.MHCK7.micro-dystrophin in Children With Duchenne Muscular Dystrophy: A Nonrandomized Controlled Trial.
Autorzy :
Mendell JR; Center for Gene Therapy, The Abigail Wexner Research Institute at Nationwide Children's Hospital, Columbus, Ohio.; Department of Pediatrics, The Ohio State University, Columbus.; Department of Neurology, The Ohio State University, Columbus.
Sahenk Z; Center for Gene Therapy, The Abigail Wexner Research Institute at Nationwide Children's Hospital, Columbus, Ohio.; Department of Pediatrics, The Ohio State University, Columbus.; Department of Neurology, The Ohio State University, Columbus.
Lehman K; Center for Gene Therapy, The Abigail Wexner Research Institute at Nationwide Children's Hospital, Columbus, Ohio.; Department of Pediatrics, The Ohio State University, Columbus.
Nease C; Center for Gene Therapy, The Abigail Wexner Research Institute at Nationwide Children's Hospital, Columbus, Ohio.; Department of Pediatrics, The Ohio State University, Columbus.
Lowes LP; Center for Gene Therapy, The Abigail Wexner Research Institute at Nationwide Children's Hospital, Columbus, Ohio.; Department of Pediatrics, The Ohio State University, Columbus.; Department of Neurology, The Ohio State University, Columbus.
Miller NF; Center for Gene Therapy, The Abigail Wexner Research Institute at Nationwide Children's Hospital, Columbus, Ohio.
Iammarino MA; Center for Gene Therapy, The Abigail Wexner Research Institute at Nationwide Children's Hospital, Columbus, Ohio.
Alfano LN; Center for Gene Therapy, The Abigail Wexner Research Institute at Nationwide Children's Hospital, Columbus, Ohio.
Nicholl A; Center for Gene Therapy, The Abigail Wexner Research Institute at Nationwide Children's Hospital, Columbus, Ohio.
Al-Zaidy S; Department of Pediatrics, The Ohio State University, Columbus.
Lewis S; Center for Gene Therapy, The Abigail Wexner Research Institute at Nationwide Children's Hospital, Columbus, Ohio.; Sarepta Therapeutics Inc, Cambridge, Massachusetts.
Church K; Center for Gene Therapy, The Abigail Wexner Research Institute at Nationwide Children's Hospital, Columbus, Ohio.
Shell R; Department of Pediatrics, The Ohio State University, Columbus.
Cripe LH; Department of Pediatrics, The Ohio State University, Columbus.
Potter RA; Center for Gene Therapy, The Abigail Wexner Research Institute at Nationwide Children's Hospital, Columbus, Ohio.; Sarepta Therapeutics Inc, Cambridge, Massachusetts.
Griffin DA; Center for Gene Therapy, The Abigail Wexner Research Institute at Nationwide Children's Hospital, Columbus, Ohio.; Sarepta Therapeutics Inc, Cambridge, Massachusetts.
Pozsgai E; Center for Gene Therapy, The Abigail Wexner Research Institute at Nationwide Children's Hospital, Columbus, Ohio.; Sarepta Therapeutics Inc, Cambridge, Massachusetts.
Dugar A; Sarepta Therapeutics Inc, Cambridge, Massachusetts.
Hogan M; Department of Radiology, Vascular and Interventional Radiology, Nationwide Children's Hospital, Columbus, Ohio.
Rodino-Klapac LR; Center for Gene Therapy, The Abigail Wexner Research Institute at Nationwide Children's Hospital, Columbus, Ohio.; Department of Pediatrics, The Ohio State University, Columbus.; Sarepta Therapeutics Inc, Cambridge, Massachusetts.
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Źródło :
JAMA neurology [JAMA Neurol] 2020 Sep 01; Vol. 77 (9), pp. 1122-1131.
Typ publikacji :
Clinical Trial, Phase I; Clinical Trial, Phase II; Controlled Clinical Trial; Journal Article; Research Support, Non-U.S. Gov't
MeSH Terms :
Dystrophin*/genetics
Outcome Assessment, Health Care*
Genetic Therapy/*methods
Muscular Dystrophy, Duchenne/*therapy
Child ; Child, Preschool ; Dependovirus ; Follow-Up Studies ; Gene Transfer Techniques ; Genetic Therapy/adverse effects ; Genetic Vectors ; Humans ; Male ; Muscle, Skeletal/metabolism ; Muscular Dystrophy, Duchenne/genetics ; Pilot Projects
Czasopismo naukowe
Tytuł :
Gene Therapy for Spinal Muscular Atrophy: Safety and Early Outcomes.
Autorzy :
Waldrop MA; Departments of Neurology and Pediatrics, The Ohio State University, Columbus, Ohio; .; Center for Gene Therapy and.
Karingada C; Departments of Neurology and.
Storey MA; Pharmacy, Nationwide Children's Hospital, Columbus, Ohio.
Powers B; Center for Gene Therapy and.
Iammarino MA; Center for Gene Therapy and.
Miller NF; Center for Gene Therapy and.
Alfano LN; Center for Gene Therapy and.
Noritz G; Department of Pediatrics, Nationwide Children's Hospital and The Ohio State University, Columbus, Ohio.
Rossman I; Departments of Pediatric Neurology and.
Ginsberg M; Departments of Pediatric Neurology and.
Mosher KA; Pediatric Physiatry, Akron Children's Hospital, Akron, Ohio.
Broomall E; Department of Pediatric Neurology, Cincinnati Children's Hospital, Cincinnati, Ohio; and.
Goldstein J; Division of Pediatric Neurology, Department of Pediatrics, Rainbow Babies and Children's Hospital, Cleveland, Ohio.
Bass N; Division of Pediatric Neurology, Department of Pediatrics, Rainbow Babies and Children's Hospital, Cleveland, Ohio.
Lowes LP; Center for Gene Therapy and.
Tsao CY; Departments of Neurology and Pediatrics, The Ohio State University, Columbus, Ohio.; Departments of Neurology and.
Mendell JR; Departments of Neurology and Pediatrics, The Ohio State University, Columbus, Ohio.; Center for Gene Therapy and.
Connolly AM; Departments of Neurology and Pediatrics, The Ohio State University, Columbus, Ohio.; Center for Gene Therapy and.
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Źródło :
Pediatrics [Pediatrics] 2020 Sep; Vol. 146 (3).
Typ publikacji :
Journal Article
MeSH Terms :
Genetic Therapy/*methods
Recombinant Fusion Proteins/*genetics
Spinal Muscular Atrophies of Childhood/*genetics
Spinal Muscular Atrophies of Childhood/*therapy
Survival of Motor Neuron 1 Protein/*genetics
Adenoviruses, Human ; Age Factors ; Alanine Transaminase/metabolism ; Aspartate Aminotransferases/metabolism ; Biological Products ; Genetic Therapy/adverse effects ; Genetic Vectors/administration & dosage ; Glucocorticoids/administration & dosage ; Humans ; Infant ; Ohio ; Outcome Assessment, Health Care ; Prednisolone/administration & dosage ; gamma-Glutamyltransferase/metabolism
Czasopismo naukowe
Tytuł :
Clinical development on the frontier: gene therapy for duchenne muscular dystrophy.
Autorzy :
Asher DR; Sarepta Therapeutics, Inc, Cambridge, Massachusetts, USA.
Thapa K; Sarepta Therapeutics, Inc, Cambridge, Massachusetts, USA.
Dharia SD; Sarepta Therapeutics, Inc, Cambridge, Massachusetts, USA.
Khan N; Sarepta Therapeutics, Inc, Cambridge, Massachusetts, USA.
Potter RA; Sarepta Therapeutics, Inc, Cambridge, Massachusetts, USA.
Rodino-Klapac LR; Sarepta Therapeutics, Inc, Cambridge, Massachusetts, USA.
Mendell JR; Center for Gene Therapy, The Abigail Wexner, Research Institute at Nationwide Children's Hospital, Columbus, Ohio, USA.; Department of Pediatrics and Neurology, The Ohio State University, Columbus, Ohio, USA.
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Źródło :
Expert opinion on biological therapy [Expert Opin Biol Ther] 2020 Mar; Vol. 20 (3), pp. 263-274. Date of Electronic Publication: 2020 Feb 12.
Typ publikacji :
Journal Article; Research Support, Non-U.S. Gov't; Review
MeSH Terms :
Dystrophin/*genetics
Muscular Dystrophy, Duchenne/*therapy
Clinical Trials as Topic ; Dependovirus/genetics ; Dystrophin/therapeutic use ; Genetic Therapy/adverse effects ; Genetic Vectors/genetics ; Genetic Vectors/metabolism ; Humans ; Muscular Dystrophy, Duchenne/genetics ; Outcome Assessment, Health Care ; Small Molecule Libraries/therapeutic use
Czasopismo naukowe

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