Autor: "Vera-Llonch M" - OpacWWW

Skocz do pozycji: 1.

Tytuł:: Symptoms and impacts of familial chylomicronemia syndrome: a qualitative study of the patient experience.
Autorzy:: Williams K; Acaster Lloyd Consulting, London, UK. .
Tickler G; Acaster Lloyd Consulting, London, UK.
Valdivielso P; Servicio de Medicina Interna, Hospital Virgen de la Victoria, University of Málaga and Instituto de Investigaciones Biomédicas de Málaga (IBIMA), Málaga, Spain.
Alonso J; IMIM-Institut Hospital del Mar d'Investigacions Mèdiques, CIBERESP, Pompeu Fabra University, Barcelona, Spain.
Vera-Llonch M; Ionis Pharmaceuticals, One Beacon Street, Boston, MA, 02120, USA.
Cubells L; Akcea Therapeutics, Avenida Ernest Lluch, 32 TCM 2 of 6.18, 08302, Mataró, Barcelona, Spain.
Acaster S; Acaster Lloyd Consulting, London, UK.; Pokaż więcej
Źródło:: Orphanet journal of rare diseases [Orphanet J Rare Dis] 2023 Oct 11; Vol. 18 (1), pp. 316. Date of Electronic Publication: 2023 Oct 11.
Typ publikacji:: Journal Article
MeSH Terms:: Hyperlipoproteinemia Type I*
Pancreatitis*/etiology
Humans ; Quality of Life ; Acute Disease ; Abdominal Pain/complications ; Patient Outcome Assessment
SCR Disease Name:: Familial hyperchylomicronemia syndrome

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Skocz do pozycji: 2.

Tytuł:: The impact of inotersen on Neuropathy Impairment Score in patients with hereditary transthyretin amyloidosis with polyneuropathy.
Autorzy:: Yarlas A; QualityMetric Incorporated, LLC, 1301 Atwood Avenue, Suite 216E, Johnston, RI, 02919, USA. .
Lovley A; QualityMetric Incorporated, LLC, 1301 Atwood Avenue, Suite 216E, Johnston, RI, 02919, USA. .
Brown D; Ionis Pharmaceuticals, Boston, MA, USA.
Vera-Llonch M; Ionis Pharmaceuticals, Boston, MA, USA.
Khella S; University of Pennsylvania, Philadelphia, PA, USA.
Karam C; University of Pennsylvania, Philadelphia, PA, USA.; Pokaż więcej
Źródło:: BMC neurology [BMC Neurol] 2023 Mar 17; Vol. 23 (1), pp. 108. Date of Electronic Publication: 2023 Mar 17.
Typ publikacji:: Clinical Trial, Phase III; Journal Article; Randomized Controlled Trial
MeSH Terms:: Amyloid Neuropathies, Familial*/complications
Amyloid Neuropathies, Familial*/drug therapy
Polyneuropathies*/diagnosis
Polyneuropathies*/drug therapy
Humans ; Muscle Weakness
SCR Disease Name:: Amyloidosis, Hereditary, Transthyretin-Related

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Skocz do pozycji: 3.

Tytuł:: Qualitative development of the PROMIS Profile v1.0-Familial Chylomicronemia Syndrome (FCS) 28.
Autorzy:: Kaiser K; Northwestern University Feinberg School of Medicine, Chicago, IL, USA. .
Fox RS; University of Arizona College of Nursing, Tucson, AZ, USA.
Perschon C; Northwestern University Feinberg School of Medicine, Chicago, IL, USA.
Vera-Llonch M; Ionis Pharmaceuticals, Carlsbad, CA, USA.
Alonso J; Hospital del Mar Medical Research Institute, Barcelona, Spain.; Universitat Pompeu Fabra, Barcelona, Spain.; CIBERESP, CIBER in Epidemiology and Public Health, Madrid, Spain.
Cubells L; Ionis Pharmaceuticals, Carlsbad, CA, USA.
Cella D; Northwestern University Feinberg School of Medicine, Chicago, IL, USA.; Pokaż więcej
Źródło:: Quality of life research : an international journal of quality of life aspects of treatment, care and rehabilitation [Qual Life Res] 2023 Feb; Vol. 32 (2), pp. 605-614. Date of Electronic Publication: 2022 Oct 31.
Typ publikacji:: Review; Journal Article
MeSH Terms:: Hyperlipoproteinemia Type I*/genetics
Hyperlipoproteinemia Type I*/diagnosis
Hyperlipoproteinemia Type I*/drug therapy
Pancreatitis*/diagnosis
Humans ; Quality of Life/psychology ; Cost of Illness
SCR Disease Name:: Familial hyperchylomicronemia syndrome

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Skocz do pozycji: 4.

Tytuł:: Early Data on Long-term Impact of Inotersen on Quality-of-Life in Patients with Hereditary Transthyretin Amyloidosis Polyneuropathy: Open-Label Extension of NEURO-TTR.
Autorzy:: Yarlas A; QualityMetric, 1301 Atwood Avenue, Suite 316E, Johnston, RI, 02919, USA. .
Lovley A; QualityMetric, 1301 Atwood Avenue, Suite 316E, Johnston, RI, 02919, USA.
McCausland K; QualityMetric, 1301 Atwood Avenue, Suite 316E, Johnston, RI, 02919, USA.
Brown D; Ionis Pharmaceuticals, Boston, MA, USA.
Vera-Llonch M; Ionis Pharmaceuticals, Boston, MA, USA.
Conceição I; Centro Hospitalar Universitário Lisboa Norte, Hospital de Santa Maria and Faculdade de Medicina, Lisbon, Portugal.
Karam C; University of Pennsylvania, Philadelphia, PA, USA.
Khella S; University of Pennsylvania, Philadelphia, PA, USA.
Obici L; Amyloidosis Research and Treatment Center, Fondazione IRCCS Policlinico San Matteo, Pavia, Italy.
Waddington-Cruz M; Federal University of Rio de Janeiro, National Amyloidosis Referral Center, CEPARM, Rio de Janeiro, Brazil.; Pokaż więcej
Źródło:: Neurology and therapy [Neurol Ther] 2021 Dec; Vol. 10 (2), pp. 865-886. Date of Electronic Publication: 2021 Aug 05.
Typ publikacji:: Journal Article

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Skocz do pozycji: 5.

Tytuł:: Rate of neuropathic progression in hereditary transthyretin amyloidosis with polyneuropathy and other peripheral neuropathies: a systematic review and meta-analysis.
Autorzy:: Lin X; QualityMetric Incorporated, LLC, Johnston, RI, USA. .
Yarlas A; QualityMetric Incorporated, LLC, Johnston, RI, USA.
Vera-Llonch M; Akcea Therapeutics, Cambridge, MA, USA.
Baranwal N; QualityMetric Incorporated, LLC, Johnston, RI, USA.
Biber J; QualityMetric Incorporated, LLC, Johnston, RI, USA.
Brown D; Akcea Therapeutics, Cambridge, MA, USA.
Vogt B; Department of Neurology, Oregon Health & Science University, Portland, OR, USA.
Karam C; Department of Neurology, Oregon Health & Science University, Portland, OR, USA.; Department of Neurology, University of Pennsylvania, Philadelphia, PA, USA.; Pokaż więcej
Źródło:: BMC neurology [BMC Neurol] 2021 Feb 12; Vol. 21 (1), pp. 70. Date of Electronic Publication: 2021 Feb 12.
Typ publikacji:: Journal Article; Meta-Analysis; Systematic Review
MeSH Terms:: Disease Progression*
Amyloid Neuropathies, Familial/*complications
Amyloid Neuropathies, Familial/*pathology
Polyneuropathies/*pathology
Female ; Humans ; Male ; Middle Aged ; Registries
SCR Disease Name:: Amyloidosis, Hereditary, Transthyretin-Related

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Skocz do pozycji: 6.

Tytuł:: The patient journey toward a diagnosis of hereditary transthyretin (ATTRv) amyloidosis.
Autorzy:: Vera-Llonch M; Global Health Economics and Outcomes, 22 Boston Wharf Road, 9th Floor, Boston, MA, 02210, USA. .
Reddy SR; Partnership for Health Analytic Research, LLC, 280 S. Beverly Dr., Ste 404, Beverly Hills, CA, 90212, USA.
Chang E; Partnership for Health Analytic Research, LLC, 280 S. Beverly Dr., Ste 404, Beverly Hills, CA, 90212, USA.
Tarbox MH; Partnership for Health Analytic Research, LLC, 280 S. Beverly Dr., Ste 404, Beverly Hills, CA, 90212, USA.
Pollock M; Global Health Economics and Outcomes, 22 Boston Wharf Road, 9th Floor, Boston, MA, 02210, USA.; Pokaż więcej
Źródło:: Orphanet journal of rare diseases [Orphanet J Rare Dis] 2021 Jan 11; Vol. 16 (1), pp. 25. Date of Electronic Publication: 2021 Jan 11.
Typ publikacji:: Journal Article; Research Support, Non-U.S. Gov't
MeSH Terms:: Amyloid Neuropathies, Familial*/diagnosis
Amyloid Neuropathies, Familial*/genetics
Prealbumin*/genetics
Aged ; Biomarkers ; Cohort Studies ; Female ; Humans ; Male ; Medicare ; Middle Aged ; United States

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Skocz do pozycji: 7.

Tytuł:: The Clinical and Economic Burden of Newly Diagnosed Hereditary Transthyretin (ATTRv) Amyloidosis: A Retrospective Analysis of Claims Data.
Autorzy:: Reddy SR; Partnership for Health Analytic Research, LLC, Beverly Hills, CA, USA.
Chang E; Partnership for Health Analytic Research, LLC, Beverly Hills, CA, USA.
Tarbox MH; Partnership for Health Analytic Research, LLC, Beverly Hills, CA, USA.
Broder MS; Partnership for Health Analytic Research, LLC, Beverly Hills, CA, USA.
Tieu RS; Partnership for Health Analytic Research, LLC, Beverly Hills, CA, USA.
Guthrie S; Akcea Therapeutics, Boston, MA, USA.
Vera-Llonch M; Akcea Therapeutics, Boston, MA, USA. .
Pollock MR; Akcea Therapeutics, Boston, MA, USA.; Pokaż więcej
Źródło:: Neurology and therapy [Neurol Ther] 2020 Dec; Vol. 9 (2), pp. 473-482. Date of Electronic Publication: 2020 May 25.
Typ publikacji:: Journal Article

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Skocz do pozycji: 8.

Tytuł:: Impact of pulmonary exacerbations and lung function on generic health-related quality of life in patients with cystic fibrosis.
Autorzy:: Solem CT; Pharmerit International, 4350 East West Hwy, Suite 430, Bethesda, MD, 20814, USA. .
Vera-Llonch M; Vertex Pharmaceuticals, 50 Northern Ave, Boston, MA, 02210, USA.
Liu S; Pharmerit International, 4350 East West Hwy, Suite 430, Bethesda, MD, 20814, USA.
Botteman M; Pharmerit International, 4350 East West Hwy, Suite 430, Bethesda, MD, 20814, USA.
Castiglione B; Vertex Pharmaceuticals, 50 Northern Ave, Boston, MA, 02210, USA.; Pokaż więcej
Źródło:: Health and quality of life outcomes [Health Qual Life Outcomes] 2016 Apr 21; Vol. 14, pp. 63. Date of Electronic Publication: 2016 Apr 21.
Typ publikacji:: Journal Article; Randomized Controlled Trial; Research Support, Non-U.S. Gov't
MeSH Terms:: Cystic Fibrosis/*physiopathology
Cystic Fibrosis/*psychology
Lung/*physiopathology
Patients/*psychology
Quality of Life/*psychology
Adolescent ; Adult ; Female ; Humans ; Male ; Respiratory Function Tests ; Young Adult

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Skocz do pozycji: 9.

Tytuł:: Effect of ivacaftor treatment in patients with cystic fibrosis and the G551D-CFTR mutation: patient-reported outcomes in the STRIVE randomized, controlled trial.
Autorzy:: Quittner A; Department of Psychology, University of Miami, 5665 Ponce de Leon Blvd, Coral Gables, FL, 33146, USA. .
Suthoff E; Vertex Pharmaceuticals Incorporated, 50 Northern Avenue, Boston, MA, 02210, USA. ellison_.
Rendas-Baum R; Optum, 24 Albion Road, Bldg 400, Lincoln, RI, 02854, USA. .
Bayliss MS; Optum, 24 Albion Road, Bldg 400, Lincoln, RI, 02854, USA. .
Sermet-Gaudelus I; Hopital Necker Enfants Malades, Universite Paris Sorbonne, 149 Rue de Sèvres, Paris, 75015, France. .
Castiglione B; Vertex Pharmaceuticals Incorporated, 50 Northern Avenue, Boston, MA, 02210, USA. brenda_.
Vera-Llonch M; Vertex Pharmaceuticals Incorporated, 50 Northern Avenue, Boston, MA, 02210, USA. montserrat_vera-llonch@vrtx.com.; Pokaż więcej
Źródło:: Health and quality of life outcomes [Health Qual Life Outcomes] 2015 Jul 02; Vol. 13, pp. 93. Date of Electronic Publication: 2015 Jul 02.
Typ publikacji:: Journal Article; Randomized Controlled Trial; Research Support, Non-U.S. Gov't
MeSH Terms:: Cystic Fibrosis Transmembrane Conductance Regulator*
Patient Outcome Assessment*
Aminophenols/*administration & dosage
Cystic Fibrosis/*drug therapy
Cystic Fibrosis/*genetics
Molecular Targeted Therapy/*methods
Quinolones/*administration & dosage
Adolescent ; Adult ; Aged ; Double-Blind Method ; Female ; Humans ; Male ; Middle Aged ; Mutation ; Quality of Life ; Young Adult