Video Blogs

Apitegromab, Scholar Rock’s muscle-directed therapy for spinal muscular atrophy (SMA), was granted a fast track designation by the U.S. Food and Drug Administration. “We are delighted to receive Fast Track designation and look forward to working closely with the FDA towards our aim of establishing apitegromab as…

Apitegromab, Scholar Rock’s muscle-directed therapy for spinal muscular atrophy (SMA), was generally safe and showed dose-dependent accumulation and clearance of the inactive form of myostatin, a muscle growth suppressor, in healthy volunteers, according to data from a Phase 1 clinical trial. These findings, published in the journal Advanced…

Apitegromab, Scholar Rock’s muscle-directed therapy for spinal muscular atrophy (SMA), safely and effectively improved or stabilized motor function in children and young adults with types 2 and 3 disease over one year, top-line data from the Phase 2 TOPAZ trial show. Notably, patients’ motor gains were either maintained or increased at…

The United States Patent and Trademark Office has granted Scholar Rock a patent for apitegromab, with add-on and combination therapies, for treating muscle conditions such as spinal muscular atrophy (SMA). “We are delighted to have been granted this unique patent that further protects our myostatin approach to…