The FDA won’t be holding an advisory panel meeting for Sarepta Therapeutics’ SRP-9001, which is now that much closer to becoming the first gene therapy for Duchenne muscular dystrophy. “2023 may be the most event eventful year in Sarepta’s event-filled history,” President and CEO Doug Ingram said on a Feb. 28...
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TORONTO, Canada — Satellos Bioscience Inc.(TSXV: MSCL) (OTCQB: MSCLF), a public biotech company developing new small molecule therapeutic approaches to improve the treatment of muscle diseases and disorders, announced today positive preliminary data showing SAT-3247 can improve skeletal muscle function in a mouse model of facioscapulohumeral muscular dystrophy (FSHD). Frank...
AUSTIN, Texas – Savara Inc. (Nasdaq: SVRA), a clinical stage biopharmaceutical company focused on rare respiratory diseases, today announced that the first patient has been dosed in the pivotal IMPALA-2 clinical trial. IMPALA-2 is a Phase 3 trial designed to evaluate the efficacy and safety of molgramostim compared to placebo. Molgramostim...
LANGHORNE, Pa — Savara Inc., a clinical stage biopharmaceutical company focused on rare respiratory diseases, recently launched aPAP ClearPath™, a new serum-based blood test that can be used by physicians in the U.S. to obtain a definitive diagnosis of aPAP, a rare autoimmune lung disease mediated by autoantibodies targeting GM-CSF....
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...
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...
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...