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Journal of Neuromuscular Diseases|February 20, 2025
A Parent Project Muscular Dystrophy-sponsored International Workshop Report on Endocrine and Bone Issues in Patients with Duchenne Muscular Dystrophy: An Ever-changing LandscapeLeanne M Ward, David R Weber, Sze Choong Wong, et al.Neurology|February 1, 2023
<i>DMD</i> Genotypes and Motor Function in Duchenne Muscular Dystrophy: A Multi-institution Meta-analysis With Implications for Clinical TrialsFrancesco Muntoni, James Signorovitch, Gautam Sajeev, et al.Lancet (London, England)|March 13, 2022
Repeated intravenous cardiosphere-derived cell therapy in late-stage Duchenne muscular dystrophy (HOPE-2): a multicentre, randomised, double-blind, placebo-controlled, phase 2 trialCraig M McDonald, Eduardo Marbán, Suzanne Hendrix, et al.Neuromuscular Disorders : NMD|June 12, 2020
Randomized phase 2 trial and open-label extension of domagrozumab in Duchenne muscular dystrophyKathryn R Wagner, Hoda Z Abdel-Hamid, Jean K Mah, et al.Journal of Neuromuscular Diseases|June 14, 2021
A Randomized, Double-Blind, Placebo-Controlled, Global Phase 3 Study of Edasalonexent in Pediatric Patients with Duchenne Muscular Dystrophy: Results of the PolarisDMD TrialRichard S Finkel, Craig M McDonald, H Lee Sweeney, et al.Journal of Neuromuscular Diseases|July 13, 2026
Navigating sexual health, fertility, and adult wellness in individuals with Duchenne muscular dystrophy: Current standards of care and future directionsNat Nasomyont, Amanda Appel, Susan Apkon, et al.Annals of Clinical and Translational Neurology|February 9, 2025
Clinical characterization of Collagen XII-related disease caused by biallelic COL12A1 variantsRiley M McCarty, Dimah Saade, Pinki Munot, et al.Muscle & Nerve|May 24, 2026
Optimizing Care for Growth and Puberty in Duchenne Muscular Dystrophy: A Survey of Clinical Practice in the OPTIMIZE DMD ConsortiumClaire L Wood, Funmbi Babalola, Robert W Benjamin, et al.Journal of the Endocrine Society|January 9, 2026
Adrenal Suppression in Duchenne Muscular Dystrophy: Management Strategies Incorporating Novel Steroid VamoroloneAnne Marie Sbrocchi, Kathi Kinnett, Maria-Elena Lautatzis, et al.Genetics in Medicine : Official Journal of the American College of Medical Genetics|November 26, 2020
An autosomal dominant neurological disorder caused by de novo variants in FAR1 resulting in uncontrolled synthesis of ether lipidsSacha Ferdinandusse, Kirsty McWalter, Heleen Te Brinke, et al.Pageof 7