Cure SMA’s year-long advocacy campaign to educate Congress and the Administration about the unmet needs of individuals with spinal muscular atrophy (SMA) has concluded with a major legislative victory. This weekend, the President signed into law a government funding bill that included a provision in support of new SMA research at the National Institutes of Health (NIH).
The SMA research provision, which was approved as part of the second FY24 Further Consolidated Appropriations Act, asks the NIH to support new SMA research to address significant unmet within the SMA community, particularly for adults with SMA.
“Cure SMA is focused on addressing the unmet needs of adults and children with SMA who lost muscle strength and motor function ability before access to an SMA treatment,” Cure SMA President Kenneth Hobby and Cure SMA Vice President for Research Jacqueline Glascock said in a joint statement. “This NIH provision is important because it means that promising basic research that Cure SMA funds will have a fair shot at being funded by the NIH to advance the research to the next phase.”
The SMA community was critical to the passage of this key SMA research provision.
Beginning in the spring of 2023, individuals with SMA and their families reached out to their Members of Congress in support of new SMA research. Through Cure SMA’s online advocacy campaign (right), hundreds of messages were sent from the SMA community asking Congress to support SMA research in the fiscal year 2024 NIH funding bill.
In addition, 115 SMA community advocates came to Washington DC as part of Cure SMA’s 2023 Hill Day to lobby for new SMA research. Adults with SMA and parents of children with SMA met with more than 140 congressional offices where they asked their U.S. Representatives and U.S. Senators to support the NIH SMA provision. And finally, Cure SMA hosted the director of the National Institute of Neurological Disorders and Stroke (NINDS) at its December 2023 Hope on the Hill event. NINDS has funded previous research that has led to the current SMA treatments.
“The voices of individuals with SMA and their families and their stories about unmet needs were heard by this Congress and President” said Maynard Friesz, Cure SMA’s Vice President for Policy and Advocacy. “This is a big team win for the SMA community that will foster new breakthroughs to address the challenges faced by children and adults with SMA.”
For more information about Cure SMA’s advocacy efforts in support of individuals with SMA and their families, go to https://www.curesma.org/cure-sma-advocacy/. To learn more about Cure SMA-funded research in SMA go to https://www.curesma.org/research/
NIH SMA RESEARCH PROVISION:
Spinal Muscular Atrophy.—The Committee remains committed to continued NIH research into spinal muscular atrophy [SMA], a neuromuscular disease that causes degenerative nerve damage and results in severe muscle loss and impaired motor function. Past SMA research at NIH, particularly through NINDS, has led to disease-modifying SMA treatments and greater knowledge of the nervous system, which has benefited other neurological and neuromuscular disorders. While current SMA treatments slow or stop future degeneration, they do not cure SMA. Individuals with SMA, particularly adults, face significant challenges in muscle weakness and fatigue due to degeneration that occurred prior to treatment. Individuals treated prior to clinical symptoms onset may also display unmet needs, such as bulbar impairment and gait abnormalities. The Committee urges NIH to address the significant unmet need that exists across all ages and disease stages of SMA by supporting new SMA research into the role and function of survival motor neuron [SMN] protein, investigation into non-SMN pathways and targets capable of modifying disease, and research into how to best combine SMN-enhancing and non-SMN approaches for optimal therapeutic outcomes.