This week, a key congressional committee recognized the unmet, everyday living needs of individuals with spinal muscular atrophy (SMA) by urging the National Institutes of Health (NIH) to support new SMA research.
The U.S. House Appropriations Committee approved a funding bill on July 10 that provides resources and direction to the NIH and other federal agencies. Thanks to the advocacy of the SMA community, the U.S House’s fiscal year 2025 bill includes a provision that directs the NIH to support new SMA research. The committee wrote that “without additional SMA research, the Committee is concerned that adults and children with SMA who were born after treatments and early diagnosis were available will continue to face chronic health challenges and significant barriers to independence.”
Cure SMA, which organized the SMA community in support of this SMA research provision, applauded the congressional action.
“Today, the voices and research needs of individuals with SMA and their families were heard,” said Maynard Friesz, Cure SMA’s Vice President for Policy and Advocacy. “Thank you to everyone in the SMA community who described their experiences, shared their stories, and advocated for new research through advocacy emails, surveys, and congressional meetings. Your advocacy mattered.”
The committee action is the first step in a year-long process that also requires approval by the full U.S. House of Representatives and the U.S. Senate.
Why is NIH funding so critical? As the world’s largest public funder of biomedical research, the NIH plays a critical role in advancing promising early research in SMA. Since 2016, the NIH has funded approximately $100 million ($8-$13 million per year) in SMA and SMA-related research. “Based on the results and learnings of seed grants funded by Cure SMA, SMA researchers next turn to the NIH, where they leverage our seed money to secure NIH research grants to further develop their SMA research, with the hope of it becoming an eventual approved treatment,” said Jacquline Glascock, PhD, Cure SMA’s Vice President for Research. Past NIH research has led to the existing SMA treatments.
Want to advocate for new SMA research and share your unmet need experience? Individuals with SMA, their families, and other supporters can inform their own Members of Congress through Cure SMA’s Advocacy Action Center about why SMA research at the NIH is so important. For more information or if you have any questions, please contact [email protected].
U.S. House Committee SMA Research Provision: (Pages 92-93)
Spinal Muscular Atrophy.—The Committee commends NIH for its continued support of research in spinal muscular atrophy (SMA), a rare neuromuscular disease. The Committee is aware that past NINDS research has led to greater understanding of the nervous system and contributed toward approved SMA treatments that slow or stop future nerve damage. The Committee also recognizes that current treatments do not cure the disease or reverse its debilitating symptoms. Without additional SMA research, the Committee is concerned that adults and children with SMA who were born after treatments and early diagnosis were available will continue to face chronic health challenges and significant barriers to independence. Furthermore, it is not yet fully known the extent of need among children treated prior to symptom onset. As such, the Committee encourages NINDS to expand its research in SMA to address fatigue, muscle weakness, motor function loss, and other unmet needs that are pervasive among the largest segment of the SMA community. Additional research into these common needs may also benefit individuals with other neuromuscular and rare diseases who face similar challenges.