Parent Project Muscular Dystrophy

Recent papers from Parent Project Muscular Dystrophy

1. Caregivers perspectives on multidisciplinary clinic visits for Duchenne and Becker muscular dystrophyJournal of Neuromuscular Diseases · 2026
2. Managing growth and puberty concerns in individuals with Duchenne muscular dystrophy: Updated guidance and future directionsJournal of Neuromuscular Diseases · 2026
3. Adrenal insufficiency in individuals with Duchenne muscular dystrophy treated with glucocorticoids: Insights from the past, current challenges, and future directionsJournal of Neuromuscular Diseases · 2026
4. Contemporary Cardiac Issues in Duchenne Muscular DystrophyCirculation · 2015
5. Functional assessments used in routine care for duchenne muscular dystrophy in the United States: A survey of certified duchenne care centersJournal of Neuromuscular Diseases · 2026
6. A structured approach to address variability in the global management of adrenal insufficiency in Duchenne muscular dystrophy: the 2025 PJ Nicholoff steroid protocolFrontiers in Neurology · 2026
7. Navigating sexual health, fertility, and adult wellness in individuals with Duchenne muscular dystrophy: Current standards of care and future directionsJournal of Neuromuscular Diseases · 2026
8. Exploring the Content Validity of Patient‐Reported Outcome Measures to Capture the Patient Experience of Becker Muscular DystrophyMuscle & Nerve · 2026
9. Care of Dystrophinopathy Carriers: Survey Results of Provider Practices in the United States (P8-9.007)Neurology · 2026
10. Optimizing Care for Growth and Puberty in Duchenne Muscular Dystrophy: A Survey of Clinical Practice in the OPTIMIZE DMD ConsortiumMuscle & Nerve · 2026
11. Meeting report: Translating exercise research in dystrophinopathy to the clinic.2026
12. TREAT-NMD advisory committee for therapeutics: Preclinical and clinical learnings from 15 years of TACTJournal of Neuromuscular Diseases · 2026
13. Motor Function and Growth Outcomes With Early Corticosteroid Initiation in Duchenne Muscular Dystrophy: An Adjusted Cross‐Trial ComparisonMuscle & Nerve · 2026
14. Heart Transplantation and Ventricular Assist Device in Duchenne Muscular Dystrophy: A New EraPediatric Transplantation · 2025
15. Re-evaluating acceptable risk of death from gene therapy: A threshold study among individuals with Duchenne muscular dystrophy and their caregivers in the US and UKEuropean Journal of Medical Genetics · 2025
16. Current Standards and Future Directions of Duchenne Muscular Dystrophy Respiratory Care: The PPMD Italy Meeting ReportPediatric Pulmonology · 2025
17. A conceptual model and practical guidance for the development, administration, and evaluation of individualized therapiesFrontiers in Medicine · 2025
18. A Parent Project Muscular Dystrophy-sponsored International Workshop Report on Endocrine and Bone Issues in Patients with Duchenne Muscular Dystrophy: An Ever-changing LandscapeJournal of Neuromuscular Diseases · 2025
19. Gaps in the Assessment and Care of Neurodevelopmental and Psychiatric Conditions Associated With DystrophinopathyMuscle & Nerve · 2024
20. Unveiling non-coding DMD variants: synergising RNA sequencing and DNA sequencing for enhanced molecular diagnosisJournal of Medical Genetics · 2024