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Rare Disease and CMD on The Hill
Cure CMD joined rare disease advocates in Washington, DC for Rare Disease Week 2026, meeting with lawmakers to raise awareness of congenital muscular dystrophy and the urgent need for research support.
James Immekus
Mar 4


FDA Announces first Platform Technology Designation: Implications for CMD’s Path to Clinical Trials
The FDA’s first Platform Technology Designation marks a major step forward for rare disease treatments, allowing reuse of key therapy components across related conditions. This breakthrough could accelerate CMD therapies, but Cure CMD needs your support to keep the momentum going.
Gustavo Dziewczapolski
Jun 27, 2025


CMD Advocacy Updates: Urge Your Senators to Reject H.R.1 Now
Learn how Cure CMD is fighting harmful legislation like the OBBBA, NIH funding cuts, and threats to Medicaid through strategic advocacy and rare disease policy action. Discover how you can help protect access to care, advance research, and make your voice heard.
Lani Knutson
Jun 25, 2025
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