Scholar Rock Presents Data Analysis of Multiple Efficacy Endpoints from the Apitegromab TOPAZ Phase 2 Trial at the American Academy of Neurology 2022 Annual Meeting
Details of the poster are as follows:
Title: Apitegromab in Spinal Muscular Atrophy (SMA): An Analysis of Multiple Efficacy Endpoints in the TOPAZ Trial (P15.005)
- The poster will present multiple efficacy endpoints, including Hammersmith scales and Revised Upper Limb Module (RULM) function, from the 12- month TOPAZ Phase 2 trial evaluating apitegromab in patients with Types 2 and 3 Spinal Muscular Atrophy (SMA).
Poster Session: P15: Child Neurology: SMA/DMD/Muscle 1
The e-poster is available live,
For conference information, visit https://www.aan.com/events
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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