SMA Press
Scholar Rock announces completion of TOPAZ trial enrollment
Scholar Rock announced today that enrollment of its TOPAZ Phase 2 trial of SRK-015 in patients with Type 2 and Type 3 SMA is complete. SRK-015 is a selective inhibitor of myostatin that has been shown to increase muscle mass and strength in animal models. For more information, please see the press release from Scholar…
Read MoreCerebrum podcast features SMA researcher Lee Rubin discussing recent advances in SMA therapeutics
SMA researcher and Harvard University professor Lee Rubin was featured in a podcast with Bill Glovin from the Dana Foundation to talk about the recent Cerebrum article he co-authored, “Spinal Muscular Atrophy: Huge Steps.” Both the article and podcast discuss recent advances in SMA therapeutics. To listen to the podcast, please visit: https://soundcloud.com/danafoundation/spinal-muscular-atrophy-huge-steps
Read MoreNovartis announces FDA filing acceptance and Priority Review of AVXS-101, a one-time treatment designed to address the genetic root cause of SMA Type 1
Basel, December 3, 2018 – Novartis today announced that the U.S. Food and Drug Administration (FDA) has accepted the company’s Biologics License Application (BLA) for AVXS-101, now known as ZOLGENSMA® (onasemnogene abeparvovec-xxxx), an investigational gene replacement therapy for the treatment of spinal muscular atrophy (SMA) Type 1. ZOLGENSMA is designed to address the genetic root cause of…
Read MoreScholar Rock Announces Publication of Preclinical Data on the Therapeutic Benefit of Inhibiting Myostatin Activation in Models of Spinal Muscular Atrophy
CAMBRIDGE, Mass., Nov. 27, 2018 (GLOBE NEWSWIRE) — Scholar Rock Holding Corporation (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 the publication, “Specific Inhibition of Myostatin Activation is Beneficial in Mouse Models of SMA Therapy” in the peer-reviewed journal Human Molecular Genetics. The publication details…
Read MoreSMA researchers Frank Bennett and Adrian Krainer win the prestigious Breakthrough Prize
Excerpted from Breakthrough Prize press release: A Total of $22 Million Awarded for Breakthroughs in Treatment for the Leading Genetic Cause of Infant Death, Super-Resolution Imaging, Discovery of a New Kind of Electronic Material, and Other Major Achievements. 2019 Breakthrough Prize in Life Sciences Awarded to C. Frank Bennett and Adrian R. Krainer. All…
Read MoreThe Spinal Muscular Atrophy Foundation Rings Nasdaq Opening Bell
The Spinal Muscular Atrophy (SMA) Foundation, a non-profit, organization whose mission is to accelerate the development of treatments for SMA, the leading cause of genetic death in young children, visited the Nasdaq MarketSite in Times Square on August 17, 2018. In honor of the occasion, Dinakar Singh, Co-Founder and Chairman of the Board of Directors,…
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