Scholar Rock Presents Exploratory Responder Analysis on Efficacy Data from the Apitegromab TOPAZ Phase 2 Trial at the Child Neurology Society Annual Meeting
Details for the Poster at the CNS meeting are as follows:
Title: 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)
- This 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.
Live poster presentation (#310) will be available on
September 30, 2021, from 11:45am- 6:30pm ETduring the exhibits and posters open session. In addition, posters will be available during the exhibits and poster review sessions with authors on September 30from 12:00-1:00pm ET, 5:30-6:30pmand October 1, 2021, from 7:00-8:00am ET.
- This poster will be published in the 2021 Annals of Neurology supplement or the CNS Annual Meeting Program
Wednesday, September 29from 6:00-7:30pm ET
Thursday, September 30from 11:30am-6:30pm ET
Friday, October 1from 7:00-10:30am ET
Apitegromab 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.
Spinal muscular atrophy (SMA) is a rare, and often fatal, genetic disorder that typically manifests in young children. An estimated 30,000 to 35,000 patients are afflicted with SMA in
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