Scholar Rock Announces that SRK-015 has Received Rare Pediatric Disease Designation from U.S. FDA for the Treatment of SMA

Aug. 13, 2020— Scholar Rock (NASDAQ: SRRK), a clinical-stage biopharmaceutical company focused on the treatment of serious diseases in which protein growth factors play a fundamental role, today announced that the U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease (RPD) designation for SRK-015 for the treatment of Spinal Muscular Atrophy (SMA), a … Fortsätt läsa Scholar Rock Announces that SRK-015 has Received Rare Pediatric Disease Designation from U.S. FDA for the Treatment of SMA

Scholar Rock Presents NEW BASE Data from the SRK-015 TOPAZ Trial

The biomarker data from the TOPAZ Phase 2 proof-of-concept trial evaluating SRK-015 for the treatment of patients with Type 2 and Type 3 Spinal Muscular Atrophy (SMA). Clinical Trials NCT03921528 Read more

TOPAZ clinical trial of SRK-015 in Type 2 and Typ 3 of Spinal Muscular Atrophy

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 … Fortsätt läsa TOPAZ clinical trial of SRK-015 in Type 2 and Typ 3 of Spinal Muscular Atrophy