#Topaz detail 3 review trial
Moderator: Thomas Crawford, M.D., lead principal investigator of the TOPAZ trial and Professor of Neurology and Pediatrics Johns Hopkins UniversityĬlinical Research Session Thursday, June 16th, 4:10 PM - 4:30 PM PST (Abstract #21)
Presenter: Hemal Shah, PharmD, an independent HEOR consultant for Scholar Rock, and CEO of Value Matters, LLC.
Title: Treatment Effects Among Patients with Type II and Type III SMA Directly Reported by Patients and Caregivers from the TOPAZ Clinical Trial “With these data and insights, we continue to build strong clinical evidence around the potential of apitegromab for the treatment of spinal muscular atrophy, a disease where significant unmet need still exists.”ĭetails of the podium presentations are as follows: “We are eager to present new data at this year’s Annual SMA Conference, in particular the 24-month efficacy and safety results from our TOPAZ trial, as well as the initial evaluation of patient treatment benefits of apitegromab on impacts to quality of life, such as social and daily living activities,” said Nagesh Mahanthappa, Ph.D., Founding Chief Executive Officer of Scholar Rock. CAMBRIDGE, Mass.-( BUSINESS WIRE)-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 company will deliver two podium presentations on 24-month data from the TOPAZ Phase 2 clinical trial at the 2022 SMA Research and Clinical Care meeting, which is being held in conjunction with Cure SMA’s Annual SMA Conference (June 15-19, 2022).
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