Phase 2 TOPAZ trial

Muscle gains with apitegromab still seen in TOPAZ, Phase 3 data on way

Should results of a Phase 3 trial of apitegromab, a muscle-directed therapy for spinal muscular atrophy, be positive when released later this year as expected — supporting the add-on therapy being approved — apitegromab could be available to U.S. patients in 2025, Scholar Rock, its developer,…

Clinical benefit of apitegromab sustained for up to 3 years: Study

Three years of treatment with apitegromab, a muscle-targeting therapy being developed by Scholar Rock, continues to provide clinical benefit to children and young adults with spinal muscular atrophy (SMA) type 2 or 3 who are not able to walk. That’s according to long-term results from the open-label…