LANGHORNE, Pa. — Savara Inc. (Nasdaq: SVRA), a clinical stage biopharmaceutical company focused on rare respiratory diseases, today announced the acceptance of two abstracts for poster presentation at the American Thoracic Society (ATS) 2024 International Conference, taking place May 17-22, 2024, in San Diego, CA. “We look forward to seeing...
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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...
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...
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...