CHARLESTON, S.C. – A South Carolina mother who has been battling a rare genetic disease for years and lost her two sons to the disease is sharing her story as MUSC works to treat this rare disease. Living in Lake City, Chantel Bass is a South Carolina resident that has...
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Yokohama, Japan – Even though psychiatric disorders such as schizophrenia, bipolar disorder, and autism spectrum disorder (ASD) are quite common, their diagnosis and treatment are challenging. While doctors today have a good idea of the clinical symptoms caused by these disorders, our overall understanding of their biological characteristics and underlying...
CLINUVEL today announced that it has reached agreement with clinical and academic experts to expand its DNA Repair Program to patients diagnosed with xeroderma pigmentosum-variant (XP-V). The Program commenced in 2020, evaluating SCENESSE® (afamelanotide 16mg) in XP-C patients.“The progression of making afamelanotide available to a further group of XP patients, who...
AMSTERDAM, the Netherlands — Scenic Biotech, a pioneer in the discovery of genetic modifiers developing therapeutics to treat severe diseases, today announced positive preclinical data for its lead small molecule QPCTL inhibitor, SC-2882. The data was also presented today by Leandro Cerchietti, MD, Richard A. Stratton Associate Professor in Hematology...
CAMBRIDGE, Mass. – Scholar Rock (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 that the United States Patent Office (USPTO) issued U.S. Patent No. 10,946,036 with an expiry of June 2037. The claims of...
CAMBRIDGE, Mass. – Scholar Rock (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 positive top-line data from the TOPAZ Phase 2 clinical trial evaluating apitegromab (SRK-015) in patients with Type 2 and Type 3 spinal muscular...
CAMBRIDGE, Mass. – Scholar Rock (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 that findings from its Phase 1 trial evaluating the safety and pharmacologic profile of apitemograb in healthy volunteers were published in the...
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...
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...
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)...