On Wednesday, June 3rd, PPMD was invited to participate in a closed roundtable with senior leadership at the U.S. Food and Drug Administration (FDA). The meeting was convened by Amy Comstock Rick, JD, Director of the Rare Disease Innovation Hub on behalf of Acting Commissioner Kyle Diamantas, JD, Acting Director of CBER, Karim Mikhail, B. Pharm, MSc, and Acting Director of CDER Michael Davis, MD, PhD.
The discussion brought together leaders from across the rare disease community and the Agency, including representatives from CDER, CBER, and the Office of Rare Disease Strategy, with PPMD representing the Duchenne voice in conversations about near- and long-term priorities for advancing rare disease innovation.
We are grateful to the FDA for continuing to create space for open dialogue and collaboration with the communities it serves.
Over the past decade, we have seen meaningful progress in Duchenne, driven in part by strong engagement and adaptable regulatory pathways from the FDA. At the same time, significant unmet need remains, particularly for individuals with rare variants who still lack viable therapeutic options.
As science continues to advance and new opportunities emerge, including earlier diagnosis through newborn screening, it is critical that regulatory pathways continue to evolve in parallel. Ensuring that innovation can reach all individuals living with Duchenne, including those with ultra-rare variants, will require continued partnership across stakeholders.
PPMD remains committed to working collaboratively with FDA, industry, and the broader rare disease community to help shape solutions that expand access and accelerate progress.
We appreciate the opportunity to ensure the Duchenne community’s perspectives were represented in this important conversation and look forward to continued engagement in the months ahead.
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