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Scholar Rock Presents Exploratory Responder Analysis on Efficacy Data from the Apitegromab TOPAZ Phase 2 Trial at the Child Neurology Society Annual Meeting
CAMBRIDGE, Mass.--(BUSINESS WIRE)-- Scholar Rock (NASDAQ: SRRK), a clinical-stage biopharmaceutical company focused on the treatment of serious diseases in
About this update from Scholar Rock Holding Corporation
[{"type":"text","content":" CAMBRIDGE, Mass.--(BUSINESS WIRE)--\nScholar 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 a late breaker poster presentation as part of the 50th Child Neurology Society (CNS) Annual Meeting taking place September 29-October 2, 2021 Virtual and Live in Boston, MA.\n\nDetails for the Poster at the CNS meeting are as follows:\n\n\nTitle: Efficacy of Apitegromab in Patients with Later-Onset Spinal Muscular Atrophy (SMA Types 2 and 3): Responder Analysis from the Phase 2 TOPAZ Study (#310)\n\n\nThis late breaking poster presentation includes exploratory responder analyses on Hammersmith scale scores, including time to achieve various thresholds of improvement in Hammersmith Functional Motor Scale Expanded (HFMSE) scores.\n\n\nLive poster presentation (#310) will be available on September 30, 2021, from 11:45am- 6:30pm ET during the exhibits and posters open session. In addition, posters will be available during the exhibits and poster review sessions with authors on September 30 from 12:00-1:00pm ET, 5:30-6:30pm and October 1, 2021, from 7:00-8:00am ET.\n\n\nThis poster will be published in the 2021 Annals of Neurology supplement or the CNS Annual Meeting Program\n\n\n\n\nScholar Rock will have a physical booth (#915) at the Hynes Convention Center, Exhibit Halls C & D, as well as a virtual booth at the meeting.\n\nLive Exhibit Hall Times:\n\n\nWednesday, September 29 from 6:00-7:30pm ET\n\n\nThursday, September 30 from 11:30am-6:30pm ET\n\n\nFriday, October 1 from 7:00-10:30am ET\n\n\nAbout Apitegromab\n\nApitegromab is a selective inhibitor of the activation of myostatin and is an investigational product candidate for the treatment of patients with spinal muscular atrophy (SMA). Myostatin, a member of the TGFβ superfamily of growth factors, is expressed primarily by skeletal muscle cells, and the absence of its gene is associated with an increase in muscle mass and strength in multiple animal species, including humans. Scholar Rock believes that inhibiting myostatin activation with apitegromab may promote a clinically meaningful improvement in motor function in patients with SMA. The U.S. Food and Drug Administration (FDA) has granted Fast Track (FTD), Orphan Drug (ODD) and Rare Pediatric Disease...