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Edgewise Therapeutics Announces Positive 2-Month Interim Results From The ARCH Open-Label Study Of EDG-5506 In Adults With Becker Muscular Dystrophy (BMD)
Significant decreases in key biomarkers of muscle damage.
CureDuchenne Ventures Commits $500,000 to 4D Molecular Therapeutics to Advance Next-Generation AAV Delivery of Gene Therapy
NEWPORT BEACH, Calif. – (BUSINESS WIRE) – CureDuchenne, a national venture philanthropy nonprofit focused on finding a cure for Duchenne muscular dystrophy (DMD), announced today a $500,000 investment into 4D […]
Crispr Halted Muscular Dystrophy In Dogs. Someday, It Might Cure Humans.
Dear Friends, Today, our partner Dr. Eric Olson and his team at Exonics, a company CureDuchenne helped form, published data in the online journal, Science that shows an increase in […]
Significant Improvements Reported in Duchenne Muscular Dystrophy Patients Treated with Capricor’s Investigational Cell Therapy
ANAHEIM, Calif., Nov. 15, 2017 /PRNewswire/ — Boys and young men in advanced stages of Duchenne muscular dystrophy experienced significant and sustained improvements in cardiac structure and function, as well […]
Exonics Therapeutics Raises $40 Million in Series A Financing
Funds to support continued development of SingleCut CRISPR technology for Duchenne muscular dystrophy CAMBRIDGE, Mass. – November 8, 2017 – Exonics Therapeutics, Inc., a biotechnology company focused on developing SingleCut CRISPR […]
Capricor Therapeutics Presents Positive Six-Month Results in Duchenne Muscular Dystrophy at World Muscle Society International Congress
SAINT MALO, France, Oct. 4, 2017 /PRNewswire/ — Capricor Therapeutics, Inc. (NASDAQ: CAPR), in its presentation today at the 22nd Annual International Congress of the World Muscle Society, reported that […]
Catabasis Pharmaceuticals Reports Positive Results from Open-Label Extension of Phase 2 MoveDMD® Trial Evaluating Edasalonexent in Duchenne Muscular Dystrophy and Plans to Initiate Phase 3 Clinical Trial in First Half 2018
CAMBRIDGE, Mass.–(BUSINESS WIRE)–Oct. 4, 2017– Catabasis Pharmaceuticals, Inc. (NASDAQ:CATB), a clinical-stage biopharmaceutical company, today reported new positive efficacy results showing sustained disease-modifying effects in the MoveDMD trial open-label extension following […]
Sarepta Therapeutics Announces its Partner, Genethon, Published New Micro-Dystrophin Gene Therapy Data in Nature Communications
Data show for the first time a systemic therapeutic effect in DMD dogs using a rAAV2/8 micro-dystrophin gene therapy approach without immunosuppressive treatment CAMBRIDGE, Mass., July 27, 2017 (GLOBE NEWSWIRE) […]
Santhera’s Raxone® Receives First Positive EAMS Scientific Opinion from UK’s MHRA in Duchenne Muscular Dystrophy
Liestal, Switzerland, June 22, 2017 – Santhera Pharmaceuticals (SIX: SANN) announces that the UK`s Medicines and Healthcare products Regulatory Agency (MHRA) has granted Raxone (idebenone) a positive scientific opinion through the Early […]