One of Cure SMA’s top priorities is to relay the SMA community’s treatment experiences and preferences to the United States Food and Drug Administration (FDA). This helps the FDA make patient-centered decisions about new SMA drugs.
2022 Cure SMA Risk/Benefit Survey Results
In the 2022 Cure SMA Risk/Benefit Survey, we asked people with SMA and their caregivers how willing they were to live with certain possible treatment risks in exchange for certain potential treatment benefits. We then compared the survey results to those from a similar 2017 survey.
The survey results showed that the SMA community is still willing to live with a range of possible treatment risks in exchange for a variety of potential treatment benefits. These findings suggest that there are still important unmet SMA treatment needs.
Cure SMA will pass on these results to the FDA to help the organization make decisions about SMA drugs that reflect the community’s current needs.
To read more about the 2022 Cure SMA Risk/Benefit Survey, visit this link.
ABOUT THE SMA INDUSTRY COLLABORATION
The Cure SMA Industry Collaboration (SMA-IC) was established in 2016 to leverage the experience, expertise, and resources of pharmaceutical and biotechnology companies, as well as other nonprofit organizations involved in the development of spinal muscular atrophy (SMA) therapeutics to more effectively address a range of scientific, clinical, and regulatory challenges. Current members include Cure SMA, Biogen, Scholar Rock, Novartis Gene Therapies, Biohaven Pharmaceuticals, Epirium Bio, Genentech/Roche, and SMA Europe. Funding for this research was provided by the 2022 SMA-IC; members include Cure SMA, Biogen, Genentech/Roche, Scholar Rock, Novartis Gene Therapies, Biohaven Pharmaceuticals, Epirium Bio, and SMA Europe.