Letter to the Editor: In response to P.R. Clemens et al., Efficacy and Safety of Viltolarsen in Boys with Duchenne Muscular Dystrophy: Results From the Phase 2, Open-Label, 4-Year Extension Study, and Long-Term Functional Efficacy and Safety of Viltolarsen in Patients with Duchenne Muscular Dystrophy-Reference-Cited by-同舟云学术

Letter to the Editor: In response to P.R. Clemens et al., Efficacy and Safety of Viltolarsen in Boys with Duchenne Muscular Dystrophy: Results From the Phase 2, Open-Label, 4-Year Extension Study, and Long-Term Functional Efficacy and Safety of Viltolarsen in Patients with Duchenne Muscular Dystrophy

Published:2023-11-07 Issue:6 Volume:10 Page:1151-1153
ISSN:2214-3599
Container-title:Journal of Neuromuscular Diseases
language:
Short-container-title:JND

Author:

Muntoni Francesco¹,Straub Volker²,Servais Laurent³,Mercuri Eugenio⁴

Affiliation:

1. Dubowitz Neuromuscular Centre and MRC Centre for Neuromuscular Diseases, UCL Great Ormond Street Institute of Child Health & Great Ormond Street Hospital for Children Foundation Trust, London, UK

2. Institute of Genetic Medicine, University of Newcastle upon Tyne, Newcastle upon Tyne, UK

3. Department of Pediatrics, University of Oxford, Oxford, UK

4. Peadiatric Neurology, Catholic University, Rome, Italy

Publisher

IOS Press

Subject

Neurology (clinical),Neurology

Reference13 articles.

1. Efficacy and Safety of Viltolarsen in Boys With Duchenne Muscular Dystrophy: Results From the Phase 2, Open-Label, 4-Year Extension Study;Clemens;J Neuromuscul Dis,2023

2. Long-Term Functional Efficacy and Safety of Viltolarsen in Patients with Duchenne Muscular Dystrophy;Clemens;J Neuromuscul Dis,2022

3. Safety, Tolerability, and Efficacy of Viltolarsen in Boys With Duchenne Muscular Dystrophy Amenable to Exon 53 Skipping: A Phase 2 Randomized Clinical Trial;Clemens;JAMA Neurol,2020

4. Suitability of external controls for drug evaluation in Duchenne muscular dystrophy;Goemans;Neurology,2020

5. Individualized Prediction of Changes in 6-Minute Walk Distance for Patients with Duchenne Muscular Dystrophy;Goemans;PLoS One,2016