To characterize patients with spinal muscular atrophy(SMA) in the AAN Axon Registry®.  

SMA was first described in the 19th century, however the genetic mutation was not discovered until the 1990’s. The first treatment was not approved until 2016, with newborn screening not recommended until 2018. While these discoveries and protocols fundamentally changed the approach to care for children born with SMA, treatment and care for the adult population remain uncertain. The Axon Registry® captures real world data (RWD) on this unique adult population for whom clinical care may be different from children.  

De-identifed Patients with SMA were manually selected in the Axon Registry if they had SMA in the opinion of the treating provider. SMA features (type, genetic copy number) were manually abstracted from EHR data contained in the Axon Registry.  Demographic, care location and treatments were extracted from structured EHR data contained in the Axon Registry. De-identified Adult SMA patients(Age ≥ 18 at time of registry entry) were compared to de-identified pediatric SMA patients.