PPMD has always fought to ensure that every individual living with Duchenne has access to safe, effective, FDA-approved treatments.
This week, PPMD joined six partner organizations: Best Day Ever Foundation, Charlie’s Cure, CureDuchenne, Little Hercules Foundation, Muscular Dystrophy Family Foundation, and Team Joseph, to urge New York State to reconsider the recent Drug Utilization Review Board (DURB) decision and to ensure coverage for ELEVIDYS in alignment with its FDA-approved label.
We respect the important role of the state’s DURB in ensuring thoughtful, evidence-based decision-making. These deliberations are critical to maintaining a fair and transparent process for evaluating new therapies. At the same time, it is essential that coverage decisions reflect the best available scientific and clinical evidence, as well as the rigorous review already conducted by federal regulatory experts. When science supports safety and efficacy, patients and families should not face additional barriers to access.
We also want to acknowledge the powerful patient and caregiver voices shared during the October 3, 2025, DURB meeting. Their stories remind us that timely access is not a luxury, it’s essential.
PPMD and our partners remain committed to working with New York and other states to ensure decisions are guided by science, compassion, and equity. We will always stand with the Duchenne community in the fight for access to approved therapies.
Access matters. Families cannot wait.
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