Cure SMA is pleased to announce the launch of the third State of SMA report. The annual data report highlights data from Cure SMA’s three databases: a patient-reported database with data from over 10,000 affected individuals worldwide that also incorporates longitudinal data from our annual Community Update Survey; the SMA Clinical Data Registry (CDR) that contains electronic medical record (EMR) sourced data from U.S. based SMA Care Center Network sites; and the Newborn Screening Registry with data from parents of babies with SMA that were identified through statewide SMA newborn screening.
The 2023 State of SMA report presents a snapshot of the quickly changing landscape of SMA. More specifically, the report includes:
- age and gender distribution of the SMA community,
- prevalence and trends of SMA type and SMN2 copy number,
- mental health,
- socioeconomic status and social determinants of health of adults with SMA,
- use of FDA approved treatments,
- unmet needs,
- patient care, and
- decreasing mortality rates
Cure SMA is thankful for all the members of the SMA community who have generously shared their data to make this report possible. Cure SMA also thanks the SMA Care Center Network for their care of patients with SMA and work with the CDR and the Care Center Network Registry Committee for their review and editing of this work. Additionally, we’re grateful for the support and funding provided by the Cure SMA Real World Evidence Collaboration (RWEC) and the Cure SMA Industry Collaboration (SMA-IC).
To view the report, please click here.
The Cure SMA Real World Evidence Collaboration
The Cure SMA Real World Evidence Collaboration (RWEC) was established in 2021 to leverage the experience, expertise and resources of pharmaceutical and biotechnology companies and nonprofit organizations involved in the development of SMA therapeutics to guide the future direction of real world evidence collection and use in SMA. Funding for the development of the State of SMA was provided by the Cure SMA RWEC. Members of the RWEC include Biogen, Genentech/Roche, and Novartis.
The Cure 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, Scholar Rock, Biogen, Novartis, Biohaven Pharmaceuticals, Genentech/Roche, Alcyone Therapeutics, NMD Pharma, and SMA Europe. Funding for the research included within the State of SMA Report was provided by the 2023 SMA-IC; members included Cure SMA, Biogen, Genentech/Roche, Scholar Rock, Novartis Gene Therapies, Biohaven Pharmaceuticals, Epirium Bio, and SMA Europe.
Cure SMA Care Center Network
Since 2018, Cure SMA has partnered with hospitals across the U.S. with the goal to improve healthcare for people with SMA. Every Care Center Network site submits consented patient information and data to the Cure SMA Clinical Data Registry. This data is then analyzed to drive healthcare improvements.