Last Updated: 09/26/2026
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Found 127 publications
Deramiocel heart-derived cellular therapy in advanced Duchenne muscular dystrophy (HOPE-3): a phase 3, randomised, double-blind, placebo-controlled trial.
Journal: Lancet (London, England)
Published: March 26, 2026
Framing childhood-onset facioscapulohumeral dystrophy: from first symptoms to future trials.
Journal: Neuromuscular disorders : NMD
Published: January 11, 2026
Natural history of limb girdle muscular dystrophy R1 (LGMDR1): a GRASP consortium study.
Journal: Neuromuscular disorders : NMD
Published: December 10, 2025
Facioscapulohumeral muscular dystrophy diagnosed in childhood: a muscular dystrophy surveillance, tracking and research network cohort.
Journal: Neuromuscular disorders : NMD
Published: October 28, 2025
Predicting Loss of Ambulation in Limb Girdle Muscular Dystrophy R9.
Journal: Annals of clinical and translational neurology
Published: October 19, 2025
279th ENMC international workshop: Classification, clinical care, outcome measures and biomarkers in childhood onset facioscapulohumeral dystrophy: towards standardizing clinical care and ensuring clinical trial readiness. Hoofddorp, The Netherlands, 1-3 November 2024.
Journal: Neuromuscular disorders : NMD
Published: September 26, 2025
Quantitative Measurement of Glycosylated ⍺-Dystroglycan as a Biomarker for Disease Severity in Limb-Girdle Muscular Dystrophy Type 2I/R9.
Journal: Neurology. Genetics
Published: June 10, 2025
Limb-Girdle Muscular Dystrophy Scientific Workshop: A Multistakeholder Discussion Focused on Charting the Path Forward for Drug Development.
Journal: Neurology. Clinical practice
Published: January 21, 2025
Clinical Trial Readiness in Limb Girdle Muscular Dystrophy R1 (LGMDR1): A GRASP Consortium Study.
Journal: Annals of clinical and translational neurology
Published: January 08, 2025
Last Updated: 09/26/2026