To further improve the health of people with SMA and extend lives, Cure SMA formed an SMA Care Center Network (CCN) and SMA registry to collect a cross section of real world data about SMA care to develop evidence-based standards of care and guidelines for SMA.

Addition of SMA to the Recommended Uniform Screening Panel facilitated pre-symptomatic  diagnosis and treatment and life altering outcomes for SMA. SMA is changing rapidly and changes in standard of care have lagged behind changes in the disease.

Neuromuscular clinics were invited to participate in the Cure SMA CCN to achieve geographic and clinic size diversity, provide multidisciplinary care, pediatric and adult care team partners, capacity to provide FDA-approved SMA therapies, and an electronic health record (EHR) informatics team supportive of EHR integration into the SMA registry. 

Each center received grant support to electronically integrate with the SMA registry. Year 1 data included 2015 Edition Common Clinical Dataset. Year 2 data included genetic testing and motor function scale(s). The Registry platform uses the Health Level Seven Fast Healthcare Interoperability Resources (FHIR) standard and employs a FHIR-based interoperability application programming interface (API) to connect to multiple EHR systems. Centers access their patient data through the SMA registry portal. 

Seventeen centers completed contracting and IRB approval. Twelve centers achieved EHR integration and are electronically sending data. 149 consented patients' data has transferred to the SMA registry. Gap analyses to identify incomplete data were shared with centers and the data platform manager to troubleshoot data transfer and optimize data quality. Data will be evaluated for outcomes and evidence to drive the SMA standard of care and guideline development.

Development of an SMA CCN and electronic transfer of EHR data to the SMA registry is feasible, scalable and the best solution to create evidence-based care guidelines and new treatment delivery.