Check Out Cure SMA’s 2024 Annual State of SMA Report
Cure SMA is pleased to announce the launch of our next State of SMA report, showcasing data through 2024. The annual data report highlights data from Cure SMA’s three databases: a patient-reported database with data from over 11,000 impacted 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 2024 State of SMA report presents a snapshot of the quickly changing landscape of SMA. More specifically, the report includes:
- The current unmet needs of those living and caring for someone with SMA
- The increase in the number of individuals with SMA living into adulthood
- Demographics and their representativeness of the SMA community
- Changing prevalence of SMA type and SMN2 copy number
- Current motor function status of children and adults living with SMA
- Social determinants of health
- The changing average age at diagnosis of SMA
- Access and use of SMA treatments
- The prevalence of depression and anxiety among the SMA community
- History of pregnancy among women living with SMA
- Access to SMA care
- Data on select comorbidities and much more
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. Current members of the RWEC include Cure SMA, 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. Funding for the research included in the State of SMA report was provided by the 2024 SMA-IC. Members of the 2024 SMA-IC include Cure SMA, Scholar Rock, Biogen, Biohaven Pharmaceuticals, Genentech/Roche, Novartis, Alcyone Therapeutics, NMD Pharma, and SMA Europe.
Cure SMA Care Center Network
The Cure SMA Care Center Network included 25 integrated SMA Care Centers that provide multidisciplinary care for people with SMA and submitted consented patient information and data to the Cure SMA Clinical Data Registry.