Cure SMA is pleased to announce the launch of its first annual State of SMA report. The purpose of this report is to share highlights from Cure SMA’s three databases: the membership database with patient reported outcomes on over 9700 affected individuals worldwide that also includes data from our annual community update survey, the SMA clinical data registry (CDR) that contains electronic medical record (EMR) data sourced from 19 US-based SMA Care Center Network sites; and the SMA newborn screening registry with data from parents of babies with SMA identified through statewide SMA newborn screening.
The 2021 State of SMA report presents a current snapshot of the quickly changing landscape of SMA. More specifically, the report includes age and gender breakdown of the SMA community; employment and education of adults affected with SMA; prevalence of SMA type and SMN2 copy number, use of FDA approved treatments, impact of newborn screening, and decreasing mortality rates. Cure SMA hopes data from this report will foster future research, programs and development of additional therapies.
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 Care Center Network for their care of patients with SMA and work with the CDR. Additionally, we’re grateful for the support and funding provided by the Cure SMA Industry Collaboration (SMA-IC), the Cure SMA Real World Evidence Collaboration (RWEC), and the Cure SMA Newborn Screening Coalition (NBSC).
To view the report, please click here.
If you are a parent of a child with SMA or an adult living with SMA and would like to share your experiences, please click on the link to complete a Community Update Survey.
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. It is currently comprised of our partners at Biogen, Genentech/Roche Pharmaceuticals, Scholar Rock, Novartis Gene Therapies, Biohaven Pharmaceuticals, Epirium Bio, and SMA Europe. Additionally, the work of the Collaboration is supported by funds provided by Biogen, Genentech/Roche, Scholar Rock, Novartis Gene Therapies, Biohaven Pharmaceuticals, and Epirium Bio.
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. Members of the RWEC include Biogen, Novartis Gene Therapies, Genentech/Roche, and SMA Europe.
The Cure SMA Newborn Screening Coalition
The Cure SMA Newborn Screening Coalition (NBSC) was established in 2017 to leverage the experience, expertise and resources of pharmaceutical and biotechnology companies and nonprofit organizations involved in the development of SMA therapeutics to advance newborn screening for SMA. Members of the NBSC include Novartis Gene Therapies, Genentech/Roche, and Biogen.
Cure SMA Care Center Network
Cure SMA has partnered with 19 SMA Care Centers across the US who provide multidisciplinary care for people with SMA. These centers are committed to establishing and implementing an evidence-based standard of care for SMA through a centralized registry collection of SMA care and treatment real world data. Consented patient electronic medical record (EMR) data is electronically transferred from the Care Center to the SMA Clinical Data Registry (CDR).