The U.S. House of Representatives recently approved legislation to renew a key U.S. Food & Drug Administration (FDA) program that has been instrumental in developing rare disease treatments, including those for spinal muscular atrophy (SMA).

The FDA's Rare Pediatric Disease Priority Review Voucher (PRV) Program was established by Congress in 2012 to spur the discovery of treatments and cures for rare diseases, such as SMA. The PRV program, along with other supportive policies, has helped drive research interest in SMA and many other rare conditions that have led to breakthrough therapies and renewed hope for individuals and families affected by rare diseases.  

The U.S. Senate must now act to renew this vital program, which expired in December 2024.

Tell your U.S. Senators to approve the Give Kids a Chance Act (H.R. 1262).

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