Selective inhibitor of the activation of myostatin (SRK-015) receives Rare Pediatric Disease Designation from US FDA for the treatment of spinal muscular atrophy
Results from an ongoing Phase II study of SRK-015 in patients with type 2 and type 3 spinal muscular atrophy (TOPAZ) are expected to be reported later in 2020.
Source:
Biospace Inc.