- The SMA landscape has changed considerably since the first reports more than a century ago of patients with spinal muscular atrophy (SMA) by Werdnig and Hofmann in 1891  and 1893 . Decoding the disease’s genetic background, first in linkage analyses [3, 4] and later by identifying mutations in SMN1 as disease-causing , paved the way for targeted medical approaches. In this review we provide an overview of both the latest therapeutic options and emerging therapies for SMA
Scholar Rock Announces the Phase 2 clinical trial of SRK-015 in TOPAZ study .
TOPAZ Phase 2 Trial Design
The Phase 2 proof-of-concept trial will evaluate the safety and efficacy of SRK-015 dosed intravenously every four weeks (Q4W) over a 12-month treatment period. The trial is anticipated to enroll approximately 55 patients with Type 2 or Type 3 SMA in the U.S., Canada, and Europe across three distinct and parallel cohorts.