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Human Mutation
|
February 15, 2022
Intron mutations and early transcription termination in Duchenne and Becker muscular dystrophy
Megan A Waldrop, Steven A Moore, Katherine D Mathews, et al.
Muscle & Nerve
|
July 6, 2023
Continued benefit of nusinersen initiated in the presymptomatic stage of spinal muscular atrophy: 5-year update of the NURTURE study
Thomas O Crawford, Kathryn J Swoboda, Darryl C De Vivo, et al.
The Lancet. Neurology
|
August 16, 2025
Safety and efficacy of apitegromab in nonambulatory type 2 or type 3 spinal muscular atrophy (SAPPHIRE): a phase 3, double-blind, randomised, placebo-controlled trial
Thomas O Crawford, Laurent Servais, Eugenio Mercuri, et al.
Muscle & Nerve
|
April 16, 2022
Randomized phase 2 study of ACE-083, a muscle-promoting agent, in facioscapulohumeral muscular dystrophy
Jeffrey M Statland, Craig Campbell, Urvi Desai, et al.
Neuromuscular Disorders : NMD
|
November 10, 2019
Nusinersen initiated in infants during the presymptomatic stage of spinal muscular atrophy: Interim efficacy and safety results from the Phase 2 NURTURE study
Darryl C De Vivo, Enrico Bertini, Kathryn J Swoboda, et al.
JCI Insight
|
March 22, 2019
A recurrent COL6A1 pseudoexon insertion causes muscular dystrophy and is effectively targeted by splice-correction therapies
Véronique Bolduc, A Reghan Foley, Herimela Solomon-Degefa, et al.
Pediatric Transplantation
|
December 22, 2025
Heart Transplantation and Ventricular Assist Device in Duchenne Muscular Dystrophy: A New Era
David N Rosenthal, Antonio Amodeo, Russell J Butterfield, et al.
Contemporary Clinical Trials
|
April 29, 2017
Developing standardized corticosteroid treatment for Duchenne muscular dystrophy
Michela Guglieri, Kate Bushby, Michael P McDermott, et al.
Neuromuscular Disorders : NMD
|
September 26, 2025
Consensus recommendations and considerations for the delivery and monitoring of gene therapy in patients with Duchenne muscular dystrophy
Jodi M Wolff, Nora Capocci, Evrim Atas, et al.
Trials
|
May 26, 2018
A checklist for clinical trials in rare disease: obstacles and anticipatory actions-lessons learned from the FOR-DMD trial
Rebecca A Crow, Kimberly A Hart, Michael P McDermott, et al.
Page
of 8
Search research articles
Search
Showing results (61-70 of 73) with videos related to
Sort By:
Page
of 8
Human Mutation
|
February 15, 2022
Intron mutations and early transcription termination in Duchenne and Becker muscular dystrophy
Megan A Waldrop, Steven A Moore, Katherine D Mathews, et al.
Muscle & Nerve
|
July 6, 2023
Continued benefit of nusinersen initiated in the presymptomatic stage of spinal muscular atrophy: 5-year update of the NURTURE study
Thomas O Crawford, Kathryn J Swoboda, Darryl C De Vivo, et al.
The Lancet. Neurology
|
August 16, 2025
Safety and efficacy of apitegromab in nonambulatory type 2 or type 3 spinal muscular atrophy (SAPPHIRE): a phase 3, double-blind, randomised, placebo-controlled trial
Thomas O Crawford, Laurent Servais, Eugenio Mercuri, et al.
Muscle & Nerve
|
April 16, 2022
Randomized phase 2 study of ACE-083, a muscle-promoting agent, in facioscapulohumeral muscular dystrophy
Jeffrey M Statland, Craig Campbell, Urvi Desai, et al.
Neuromuscular Disorders : NMD
|
November 10, 2019
Nusinersen initiated in infants during the presymptomatic stage of spinal muscular atrophy: Interim efficacy and safety results from the Phase 2 NURTURE study
Darryl C De Vivo, Enrico Bertini, Kathryn J Swoboda, et al.
JCI Insight
|
March 22, 2019
A recurrent COL6A1 pseudoexon insertion causes muscular dystrophy and is effectively targeted by splice-correction therapies
Véronique Bolduc, A Reghan Foley, Herimela Solomon-Degefa, et al.
Pediatric Transplantation
|
December 22, 2025
Heart Transplantation and Ventricular Assist Device in Duchenne Muscular Dystrophy: A New Era
David N Rosenthal, Antonio Amodeo, Russell J Butterfield, et al.
Contemporary Clinical Trials
|
April 29, 2017
Developing standardized corticosteroid treatment for Duchenne muscular dystrophy
Michela Guglieri, Kate Bushby, Michael P McDermott, et al.
Neuromuscular Disorders : NMD
|
September 26, 2025
Consensus recommendations and considerations for the delivery and monitoring of gene therapy in patients with Duchenne muscular dystrophy
Jodi M Wolff, Nora Capocci, Evrim Atas, et al.
Trials
|
May 26, 2018
A checklist for clinical trials in rare disease: obstacles and anticipatory actions-lessons learned from the FOR-DMD trial
Rebecca A Crow, Kimberly A Hart, Michael P McDermott, et al.
Page
of 8