SMA

Sep 18, 2026

Summary The FDA approved Scholar Rock’s ISEMBYLD on September 11, 2026, the first and only muscle-targeted therapy for SMA, for adults and children aged 2+ who are already on an SMN2-targeted treatment.  The FDA approval was based on the Phase 3 SAPPHIRE trial, which enrolled 188 patients aged 2–21 with 5q S...

Aug 30, 2019

Spinal Muscular Atrophy is a rare and the second most fatal autosomal recessive genetic disorder with an incidence of 1 in 6000 to 1 in 10,000 births. Globally talking, SMA prevalence was reported in 19,343 cases in the year 2017 in the 7MM, and the US accounted for the highest number of cases approximately around ...

Aug 11, 2016

Spinal Muscular Atrophy (SMA) is a rare genetic and neuromuscular condition which affects mainly children. It has several distinct types such as type 1 (Werdnig-Hoffmann disease), type 2 (Dubowitz disease), type 3 (Kugelberg-Welander disease) and type 4 (Adult onset). SMN1 (Survival Motor Neuron 1) is the primary ge...