FAMILIES OF SPINAL MUSCULAR ATROPHY

Cure sma focuses on three different yet equally critical and interdependent research areas: basic research, to understand the cause and biology of sma; drug discovery, to convert basic research ideas into practical new drug candidates; and clinical & regulatory research, to provide the infrastructure to test the drug candidates. During this period cure sma funded six new and six ongoing basic research grants. We also funded and executed multiple clinical and regulatory focused projects, to increase sma clinical trial site capacity and readiness, to provide regulatory authorities with data on the sma population and their reported unmet needs, and to collect data to understand the patient experience with sma. Currently, there are three approved drugs for sma, with another several dozen drug programs in development, including five in clinical trials. Currently, two investigational drugs are awaiting fda approval.cure sma have three primary databases for data collection: 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 above data sources are combined and analyzed annually to identify trends in demographics, diagnosis, sma treatments, unmet needs, patient care and outcomes. The most recent analyzes were published in the state of sma 2024 report available on our website. Data collected is also shared in peer-reviewed literature. Cure sma published "sma community priorities for future drug therapies: cross-sectional survey finding representing 410 adults with sma" in neurology and therapy in june 2025. In addition, cure sma supports the sma industry collaboration. Established in 2016, the collaboration is a multi-faceted partnership that brings together pharmaceutical companies, cure sma, and other nonprofit organizations to share information, ideas, and data. The sma industry collaboration works together to address scientific, clinical, and regulatory topics that are critical to advancing drug development in sma and will benefit the broader sma community. Cure sma co-created a new healthcare provider enduring continuing medical education program in addition to the in-person cure sma clinical care session offered in june 2025. Resources were also devoted to maintaining website listings of sma newborn screening referral centers and treatment centers that provide fda-approved sma treatments.