CAMBRIDGE, Mass. – Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today announced positive 12-week expression and safety results from the first 11 participants enrolled in Study SRP-9001-103, an open-label study known as ENDEAVOR being conducted in partnership with Roche. In results from the first...
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CAMBRIDGE, Mass. – Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today shared new of SRP-9003 (rAAVrh74.MHCK7.hSGCB), the Company’s investigational gene therapy for limb-girdle muscular dystrophy Type 2E (LGMD2E). In the first look at expression data from biopsies taken two years after a single administration...
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...
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...