CAMBRIDGE, Mass. — Scholar Rock (NASDAQ: SRRK), a global biopharmaceutical company dedicated to improving the lives of children and adults with spinal muscular atrophy (SMA) and additional rare, severe, and debilitating neuromuscular diseases by applying its leading platform in myostatin biology to advance musculoskeletal health, today announced it has resubmitted...
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CAMBRIDGE, Mass. — Scholar Rock (NASDAQ: SRRK), a global biopharmaceutical company dedicated to improving the lives of patients with rare, severe, and debilitating neuromuscular diseases by applying its world-leading platform in myostatin biology, today provided an update on the anticipated timing of a Committee for Medicinal Products for Human Use (CHMP)...
Oral presentation on the positive Phase 3 SAPPHIRE trial of apitegromab for people living with spinal muscular atrophy (SMA), which demonstrated statistically significant improvement and clinically meaningful benefit as measured by the Hammersmith Functional Motor Scale Expanded (HFMSE) Apitegromab Biologics License Application (BLA) accepted under priority review for patients with...
CAMBRIDGE, Mass. — Scholar Rock (NASDAQ: SRRK), a global biopharmaceutical company dedicated to improving the lives of patients with rare, severe, and debilitating neuromuscular diseases by applying its world-leading platform in myostatin biology, today announced that the FDA has granted Fast Track and Orphan Drug designations to apitegromab for the...
CAMBRIDGE, Mass. — Scholar Rock (NASDAQ: SRRK), a late-stage biopharmaceutical company focused on developing and commercializing apitegromab for children and adults with spinal muscular atrophy (SMA) and other severe and debilitating neuromuscular diseases, announced today that positive study results from the pivotal Phase 3 SAPPHIRE trial (NCT05156320) were published in the peer-reviewed journal The...
CAMBRIDGE, Mass. — Scholar Rock (NASDAQ: SRRK), a global biopharmaceutical company dedicated to improving the lives of patients with rare, severe, and debilitating neuromuscular diseases by applying its leading platform in myostatin biology, today announced that the FDA has approved ISEMBYLD (apitegromab-mstn) for the treatment of spinal muscular atrophy (SMA)...
Demonstrates the potential of SGR-1505 as a novel approach for diseases with high unmet medical need NEW YORK, NY — Schrödinger, Inc. (Nasdaq: SDGR) today announced that SGR-1505, its clinical stage MALT1 inhibitor, was designated as a Fast Track product by the U.S. Food and Drug Administration (FDA) for the...
LOS ANGELES – Science 37, the industry leader in decentralized clinical trials, announced today a partnership with Xperiome, a global rare disease healthtech company. The partnership further strengthens Science 37’s capabilities to bring research directly to patients, both by easing the patient burden and enabling treating physicians the opportunity to...
Swindon, UK – Researchers have shown for the first time that a crucial interface in a protein that drives cancer growth could act as a target for more effective treatments. The study was led by the Science and Technology Facilities Council (STFC) Central Laser Facility (CLF) and used advanced laser...
BOSTON, Mass.c – In a paper published in the Journal of the American Medical Association (JAMA), neurologists at Beth Israel Deaconess Medical Center (BIDMC) showed that a simple skin biopsy test detects an abnormal form of alpha-synuclein, the pathological hallmark of Parkinson’s disease and the subgroup of neurodegenerative disorders known...