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Journal of Comparative Effectiveness Research
|
March 25, 2026
Ataluren for the treatment of people living with nonsense mutation Duchenne muscular dystrophy: a plain language summary of Study 041
Shiwen Wu, Dmitry Vlodavets, Alexandra Prufer de Queiroz Campos Araujo, et al.
Journal of Neuromuscular Diseases
|
November 18, 2016
Skeletal Muscle Quantitative Nuclear Magnetic Resonance Imaging and Spectroscopy as an Outcome Measure for Clinical Trials
Pierre G Carlier, Benjamin Marty, Olivier Scheidegger, et al.
International Journal of Neonatal Screening
|
May 23, 2023
Pilot Program of Newborn Screening for 5q Spinal Muscular Atrophy in the Russian Federation
Kristina Mikhalchuk, Olga Shchagina, Alena Chukhrova, et al.
Orphanet Journal of Rare Diseases
|
July 29, 2022
Natural history of Type 1 spinal muscular atrophy: a retrospective, global, multicenter study
Claude Cances, Dmitry Vlodavets, Giacomo Pietro Comi, et al.
The New England Journal of Medicine
|
July 28, 2021
Risdiplam-Treated Infants with Type 1 Spinal Muscular Atrophy versus Historical Controls
Basil T Darras, Riccardo Masson, Maria Mazurkiewicz-Bełdzińska, et al.
The Lancet. Neurology
|
October 16, 2022
Safety and efficacy of risdiplam in patients with type 1 spinal muscular atrophy (FIREFISH part 2): secondary analyses from an open-label trial
Riccardo Masson, Maria Mazurkiewicz-Bełdzińska, Kristy Rose, et al.
International Journal of Molecular Sciences
|
October 16, 2025
Balanced Translocations Involving the <i>DMD</i> Gene as a Cause of Muscular Dystrophy in Female Children: A Description of Three Cases
Ekaterina O Vorontsova, Aysylu Murtazina, Elena Zinina, et al.
The New England Journal of Medicine
|
August 13, 2025
Risdiplam in Presymptomatic Spinal Muscular Atrophy
Richard S Finkel, Laurent Servais, Dmitry Vlodavets, et al.
Journal of Comparative Effectiveness Research
|
August 22, 2025
Confirmatory long-term efficacy and safety results of ataluren in patients with nmDMD from Study 041, an international, randomized, double-blind, placebo-controlled, Phase III trial
Dmitry Vlodavets, Shiwen Wu, Anna Kostera-Pruszczyk, et al.
Journal of Neurology, Neurosurgery, and Psychiatry
|
September 11, 2017
MRI in sarcoglycanopathies: a large international cohort study
Giorgio Tasca, Mauro Monforte, Jordi Díaz-Manera, et al.
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of 2
Search research articles
Search
Showing results (1-10 of 12) with videos related to
Sort By:
Page
of 2
Journal of Comparative Effectiveness Research
|
March 25, 2026
Ataluren for the treatment of people living with nonsense mutation Duchenne muscular dystrophy: a plain language summary of Study 041
Shiwen Wu, Dmitry Vlodavets, Alexandra Prufer de Queiroz Campos Araujo, et al.
Journal of Neuromuscular Diseases
|
November 18, 2016
Skeletal Muscle Quantitative Nuclear Magnetic Resonance Imaging and Spectroscopy as an Outcome Measure for Clinical Trials
Pierre G Carlier, Benjamin Marty, Olivier Scheidegger, et al.
International Journal of Neonatal Screening
|
May 23, 2023
Pilot Program of Newborn Screening for 5q Spinal Muscular Atrophy in the Russian Federation
Kristina Mikhalchuk, Olga Shchagina, Alena Chukhrova, et al.
Orphanet Journal of Rare Diseases
|
July 29, 2022
Natural history of Type 1 spinal muscular atrophy: a retrospective, global, multicenter study
Claude Cances, Dmitry Vlodavets, Giacomo Pietro Comi, et al.
The New England Journal of Medicine
|
July 28, 2021
Risdiplam-Treated Infants with Type 1 Spinal Muscular Atrophy versus Historical Controls
Basil T Darras, Riccardo Masson, Maria Mazurkiewicz-Bełdzińska, et al.
The Lancet. Neurology
|
October 16, 2022
Safety and efficacy of risdiplam in patients with type 1 spinal muscular atrophy (FIREFISH part 2): secondary analyses from an open-label trial
Riccardo Masson, Maria Mazurkiewicz-Bełdzińska, Kristy Rose, et al.
International Journal of Molecular Sciences
|
October 16, 2025
Balanced Translocations Involving the <i>DMD</i> Gene as a Cause of Muscular Dystrophy in Female Children: A Description of Three Cases
Ekaterina O Vorontsova, Aysylu Murtazina, Elena Zinina, et al.
The New England Journal of Medicine
|
August 13, 2025
Risdiplam in Presymptomatic Spinal Muscular Atrophy
Richard S Finkel, Laurent Servais, Dmitry Vlodavets, et al.
Journal of Comparative Effectiveness Research
|
August 22, 2025
Confirmatory long-term efficacy and safety results of ataluren in patients with nmDMD from Study 041, an international, randomized, double-blind, placebo-controlled, Phase III trial
Dmitry Vlodavets, Shiwen Wu, Anna Kostera-Pruszczyk, et al.
Journal of Neurology, Neurosurgery, and Psychiatry
|
September 11, 2017
MRI in sarcoglycanopathies: a large international cohort study
Giorgio Tasca, Mauro Monforte, Jordi Díaz-Manera, et al.
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of 2