Scholar Rock will present new Phase 3 SAPPHIRE trial data on apitegromab at the 2025 MDA conference, highlighting efficacy ...

Data from the SAPPHIRE study formed the basis of Scholar Rock's U.S. Food and Drug Administration request to approve ...

Scholar Rock (SRRK) announced that new efficacy and safety data from the Phase 3 pivotal SAPPHIRE trial will be presented in multiple clinical ...

A child with a rare genetic disorder — spinal muscular atrophy type I — was treated prenatally for the first time. Courtesy of Jakob Owens  via Unsplash During an amniocentesis test, a long, thin ...

The tests revealed that the fetus also had mutations in the SMN1 genes in both chromosomes. The team intervened at 32-week gestation period by giving the mother risdiplam - a drug approved by the ...