Myotonic Dystrophy Foundation

Recent papers from Myotonic Dystrophy Foundation

1. From consent to participatory governance: The need for multi-stakeholder alignment in iPSC-based drug repurposingStem Cell Reports · 2026
2. Myotonic dystrophy family registry. The patient experienceJournal of Neuromuscular Diseases · 2026
3. Dreams, dollars, and donors: organizational actorhood and the rising development orientation of global higher educationSocial Forces · 2026
4. Evaluation of the methodology of independent Community Advisory Boards in health products research and development: a mixed-methods cross-sectional survey studyResearch Involvement and Engagement · 2026
5. Myotonic Dystrophy ( DM ) Burden of Disease: A Retrospective Study of Healthcare Costs and Utilization by Individuals With DMMuscle & Nerve · 2025
6. International collaboration to improve knowledge on myotonic dystrophy type 2Journal of Neuromuscular Diseases · 2024
7. A Five-Year Review of Newborn Screening for Spinal Muscular Atrophy in the State of Utah: Lessons LearnedInternational Journal of Neonatal Screening · 2024
8. Enhancing evidence-informed policymaking in medicine and healthcare: stakeholder involvement in the Commons Project for rare diseases in JapanResearch Involvement and Engagement · 2023
9. Healthcare resource utilization, total costs, and comorbidities among patients with myotonic dystrophy using U.S. insurance claims data from 2012 to 2019Orphanet Journal of Rare Diseases · 2022
10. Clinical Care Recommendations for Cardiologists Treating Adults With Myotonic DystrophyJournal of the American Heart Association · 2020
11. Patient Input to Inform the Development of Central Nervous System Outcome Measures in Myotonic DystrophyTherapeutic Innovation & Regulatory Science · 2020
12. Consensus-Based Care Recommendations for Pulmonologists Treating Adults with Myotonic Dystrophy Type 1Respiration · 2020
13. Consensus-based care recommendations for congenital and childhood-onset myotonic dystrophy type 1Neurology Clinical Practice · 2019
14. Seeking a better landscape for therapy development in neuromuscular disordersMuscle & Nerve · 2017
15. The Impact of Pregnancy on Myotonic Dystrophy: A Registry-Based StudyJournal of Neuromuscular Diseases · 2015
16. Congenital myotonic dystrophy in a national registryPaediatrics & Child Health · 2010