CAMBRIDGE, Mass. – Scholar Rock (NASDAQ: SRRK), a late-stage biopharmaceutical company focused on advancing innovative treatments for neuromuscular diseases, cardiometabolic disorders, and other serious diseases where protein growth factors play a fundamental role, today announced that new efficacy and safety data from the Phase 3 pivotal SAPPHIRE trial (NCT05156320) will be...
treatment News
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...
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 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...
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...
Boca Raton, FL – Extracted from the core of sandalwood trees (santalum album tree), sandalwood oil has been used for many centuries by several cultures throughout the world for perfume, soaps, incense and candles. With its earthy sweet scent, this essential oil also is used in the food industry and...
San Francisco, Calif. – Researchers have newly identified a universal, essential biomarker for the childhood cancer neuroblastoma – and a potential new target for treatment. Neuroblastoma accounts for 15% of all pediatric cancer deaths and is the most common source of childhood tumors outside of brain cancer. The disease develops...
Beijing, China – Researchers from the Institute of Process Engineering (IPE) of the Chinese Academy of Sciences and Beijing Chaoyang Hospital have developed a new “pseudo cell” formulation based on self-healing microcapsule-loading exosomes to treat diverse vitreoretinal diseases. Vitreoretinal diseases include a wide spectrum of vision-threatening disorders, which may cause...
Plymouth, England – Scientists have developed a potentially transformative new technique that could aid in the discovery and development of new therapeutics for a number of globally prevalent autoimmune diseases. Conditions such as lupus, rheumatoid arthritis and inflammatory bowel disease (IBD) – as well as failures within transplanted cells –...
Sacramento, CA – Researchers at UC Davis Comprehensive Cancer Center have shown that inhibiting a specific protein using gene therapy can shrink hepatocellular carcinoma (HCC) in mice. Silencing the galectin 1 (Gal1) protein, which is often over-expressed in HCC, also improved the anti-cancer immune response and increased the number of killer T cells inside tumors....