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...
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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...
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...
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...