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-Reference-Cited by-同舟云学术

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

Published:2021-04 Issue:4 Volume:20 Page:284-293
ISSN:1474-4422
Container-title:The Lancet Neurology
language:en
Short-container-title:The Lancet Neurology

Author:

Day John W,Finkel Richard S,Chiriboga Claudia A,Connolly Anne M,Crawford Thomas O,Darras Basil T,Iannaccone Susan T,Kuntz Nancy L,Peña Loren D M,Shieh Perry B,Smith Edward C,Kwon Jennifer M,Zaidman Craig M,Schultz Meredith,Feltner Douglas E,Tauscher-Wisniewski Sitra,Ouyang Haojun,Chand Deepa H,Sproule Douglas M,Macek Thomas A,Mendell Jerry R

Publisher

Elsevier BV

Subject

Clinical Neurology

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3. Quantitative analyses of SMN1 and SMN2 based on real-time lightCycler PCR: fast and highly reliable carrier testing and prediction of severity of spinal muscular atrophy;Feldkötter;Am J Hum Genet,2002

4. Observational study of spinal muscular atrophy type I and implications for clinical trials;Finkel;Neurology,2014

5. Natural history of infantile-onset spinal muscular atrophy;Kolb;Ann Neurol,2017

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1. Outcomes for patients in the RESTORE registry with spinal muscular atrophy and four or more SMN2 gene copies treated with onasemnogene abeparvovec;European Journal of Paediatric Neurology;2024-11

2. Real-world multidisciplinary outcomes of onasemnogene abeparvovec monotherapy in patients with spinal muscular atrophy type 1: experience of the French cohort in the first three years of treatment;Orphanet Journal of Rare Diseases;2024-09-13

3. Thrombospondin-4 as potential cerebrospinal fluid biomarker for therapy response in pediatric spinal muscular atrophy;Journal of Neurology;2024-09-06

4. Meeting Report: 2023 Muscular Dystrophy Association Summit on ‘Safety and Challenges in Gene Therapy of Neuromuscular Diseases’;Journal of Neuromuscular Diseases;2024-09-03

5. A Systematic Literature Review of the Natural History of Respiratory, Swallowing, Feeding, and Speech Functions in Spinal Muscular Atrophy (SMA);Journal of Neuromuscular Diseases;2024-09-03