Scholar Rock to Present New 24-Month Data from Phase 2 TOPAZ Study at Two International Conferences: World Muscle Society Congress and International Scientific Congress on Spinal Muscular Atrophy
“These findings provide further evidence of the potential of apitegromab, which is the first and only muscle-directed myostatin therapy to show positive proof of concept in SMA,” said
Details of the presentations are as follows:
Title: Apitegromab in SMA: An analysis of multiple efficacy endpoints in the TOPAZ extension study (P.102)
Presentation Type: Poster Session 2
Date and Time:
Title: Apitegromab in SMA: Efficacy, Safety and PK/PD Assessments in 24 Months of TOPAZ
Presentation Type: Oral
Date and Time:
The presentations will be made available in the Publications & Posters section of Scholar Rock’s website following the conferences.
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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