Cure SMA Database Findings Published in the Journal of Neuromuscular Diseases
An important goal at Cure SMA is to collect data to better understand the SMA patient population, and then to report this data back to our industry partners, regulators, and payers. In collecting this data, we also hope to identify areas where additional care, advocacy, support, and research are needed. To help accomplish this goal, Cure SMA has been collecting patient-reported data through various surveys, projects, and personal outreach. In March 2018 an article written by both Cure SMA team members and external SMA researchers on the description of the Cure SMA database was published in the Journal of Neuromuscular Diseases. Publishing in a peer-reviewed scientific journal is critical to our dissemination process. This type of journal publishing adds credibility to the data, because it is vetted by experts and quite competitive. Furthermore, the publication of this article will allow other scientists to review and utilize the results as the foundation for their research.
The article, “An overview of the Cure SMA membership database: Highlights of key demographic and clinical characteristics of SMA members” described 1,966 affected individuals who reached out to Cure SMA between 2010 and 2016 from around the world. The majority of the affected individuals (51.9%) had type I SMA, followed by type II SMA (32.3%) and then type III SMA (15.8%). The article further described the average age of SMA diagnosis as 5.2 months for type I, 22.1 months for type II, and 97.8 months for type III. The median time to reach out to Cure SMA following a diagnosis is 2 weeks for type I and 3 weeks for both type II and type III. According to the database, the median survival of those affected with type I SMA is 13.6 months which is increasing from earlier cohorts both in the database and published literature. Lastly, and most importantly, the Cure SMA database proves to be a rich resource for SMA research as it is estimated to capture about 60% of all affected individuals with SMA in the United States based on an estimated birth prevalence of 1 in 11,000.
Cure SMA would like to thank all the authors on their contribution and accomplishment on the publication of this article. And more importantly, Cure SMA would like to thank the entire SMA community for sharing their stories as all of their information is helping SMA research more forward.