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Pediatric Clinics of North America
|
July 6, 2025
Hypotonia in the Newborn Infant
Chamindra G Laverty
Advances in Therapy
|
November 11, 2024
Assessment of Phosphorodiamidate Morpholino Oligomer Treatment Patterns for Patients with Duchenne Muscular Dystrophy: A MarketScan Claims Analysis
Alexa C Klimchak, James Signorovitch, Bryan Innis, et al.
ERJ Open Research
|
September 27, 2023
Effectiveness of long-term noninvasive ventilation measured by remote monitoring in neuromuscular disease
Jeremy E Orr, Kenneth Chen, Florin Vaida, et al.
Journal of Multidisciplinary Healthcare
|
September 3, 2024
Healthcare Stakeholder Perspectives on a Value Assessment Approach for Duchenne Muscular Dystrophy Therapies
Ryan Fischer, Pat Furlong, Annie Kennedy, et al.
Journal of Neuromuscular Diseases
|
November 10, 2025
Onasemnogene abeparvovec gene therapy for treatment of patients with spinal muscular atrophy: Updated real-world practical considerations
Crystal M Proud, Elizabeth A Kichula, Susan E Matesanz, et al.
HGG Advances
|
February 15, 2024
Clinical, immunohistochemical, and genetic characterization of splice-altering biallelic DES variants: Therapeutic implications
Janelle Geist Hauserman, Chamindra G Laverty, Sandra Donkervoort, et al.
Neurology
|
October 28, 2025
Expert Consensus on Genetic Diagnostic Approaches for Patients With Limb-Girdle Muscular Dystrophy
Volker Straub, Amanda R Clause, Sandra Donkervoort, et al.
JCI Insight
|
July 30, 2024
Missense variants in CMS22 patients reveal that PREPL has both enzymatic and nonenzymatic functions
Yenthe Monnens, Anastasia Theodoropoulou, Karen Rosier, et al.
The Lancet. Neurology
|
March 20, 2024
Safety and efficacy of givinostat in boys with Duchenne muscular dystrophy (EPIDYS): a multicentre, randomised, double-blind, placebo-controlled, phase 3 trial
Eugenio Mercuri, Juan J Vilchez, Odile Boespflug-Tanguy, et al.
Annals of Clinical and Translational Neurology
|
February 9, 2025
Clinical characterization of Collagen XII-related disease caused by biallelic COL12A1 variants
Riley M McCarty, Dimah Saade, Pinki Munot, 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
Pediatric Clinics of North America
|
July 6, 2025
Hypotonia in the Newborn Infant
Chamindra G Laverty
Advances in Therapy
|
November 11, 2024
Assessment of Phosphorodiamidate Morpholino Oligomer Treatment Patterns for Patients with Duchenne Muscular Dystrophy: A MarketScan Claims Analysis
Alexa C Klimchak, James Signorovitch, Bryan Innis, et al.
ERJ Open Research
|
September 27, 2023
Effectiveness of long-term noninvasive ventilation measured by remote monitoring in neuromuscular disease
Jeremy E Orr, Kenneth Chen, Florin Vaida, et al.
Journal of Multidisciplinary Healthcare
|
September 3, 2024
Healthcare Stakeholder Perspectives on a Value Assessment Approach for Duchenne Muscular Dystrophy Therapies
Ryan Fischer, Pat Furlong, Annie Kennedy, et al.
Journal of Neuromuscular Diseases
|
November 10, 2025
Onasemnogene abeparvovec gene therapy for treatment of patients with spinal muscular atrophy: Updated real-world practical considerations
Crystal M Proud, Elizabeth A Kichula, Susan E Matesanz, et al.
HGG Advances
|
February 15, 2024
Clinical, immunohistochemical, and genetic characterization of splice-altering biallelic DES variants: Therapeutic implications
Janelle Geist Hauserman, Chamindra G Laverty, Sandra Donkervoort, et al.
Neurology
|
October 28, 2025
Expert Consensus on Genetic Diagnostic Approaches for Patients With Limb-Girdle Muscular Dystrophy
Volker Straub, Amanda R Clause, Sandra Donkervoort, et al.
JCI Insight
|
July 30, 2024
Missense variants in CMS22 patients reveal that PREPL has both enzymatic and nonenzymatic functions
Yenthe Monnens, Anastasia Theodoropoulou, Karen Rosier, et al.
The Lancet. Neurology
|
March 20, 2024
Safety and efficacy of givinostat in boys with Duchenne muscular dystrophy (EPIDYS): a multicentre, randomised, double-blind, placebo-controlled, phase 3 trial
Eugenio Mercuri, Juan J Vilchez, Odile Boespflug-Tanguy, et al.
Annals of Clinical and Translational Neurology
|
February 9, 2025
Clinical characterization of Collagen XII-related disease caused by biallelic COL12A1 variants
Riley M McCarty, Dimah Saade, Pinki Munot, et al.
Page
of 2