Jesse's Journey Foundation

– Pivotal Study of Novel Inhibitor of NF-kB, a Key Driver of Skeletal Muscle Disease and Cardiomyopathy in Duchenne — CAMBRIDGE, Mass.–(BUSINESS WIRE)–Catabasis Pharmaceuticals, Inc. (NASDAQ:CATB), a clinical-stage biopharmaceutical company, today announced the initiation of PolarisDMD, the Company’s Phase 3 trial for edasalonexent in Duchenne muscular dystrophy (DMD). Edasalonexent inhibits NF-kB, which is the key link […]

Sarepta’s early-stage clinical trial for a gene therapy for Duchenne muscular dystrophy is back on schedule, the Cambridge biotech said Monday. The Food and Drug Administration had put the trial on hold in July. The FDA ordered the hold after small fragments of DNA turned up in the treatment, which is being used at Nationwide […]

Vamorolone, a Duchenne muscular dystrophy (DMD) treatment candidate, showed anti-inflammatory activity and an improved safety profile over the glucocorticoid prednisolone in a Phase 2a clinical trial for boys 4 to 6 years old. Findings from the trial were published in the study, “Phase IIa trial in Duchenne muscular dystrophy shows vamorolone is a first-in-class dissociative steroidal anti-inflammatory […]

— Relying upon Scientific Advisory Group input, Sarepta will seek further scientific advice from European Medicines Agency on a possible path to bring eteplirsen to patients in Europe — CAMBRIDGE, Mass., September 21, 2018 (GLOBE NEWSWIRE) – Sarepta Therapeutics, Inc. (NASDAQ: SRPT), a commercial-stage biopharmaceutical company focused on the discovery and development of precision genetic […]

Researchers used CRISPR gene editing successfully in a large animal to repair DNA mutations Listen Here A team of researchers may be one step closer to curing a devastating form of muscular dystrophy, after announcing they successfully used gene editing to stop the progression of the disease in dogs. “To say it was exhilarating to see the […]

DALLAS – Aug. 30, 2018 – Scientists for the first time have used CRISPR gene editing to halt the progression of Duchenne muscular dystrophy (DMD) in a large mammal, according to a study by UT Southwestern that provides a strong indication that a lifesaving treatment may be in the pipeline. The research published in Sciencedocuments unprecedented […]

AbCellera Biologics Inc. has announced a collaboration with Fabio Rossi, MD, PhD, and Michael Underhill, PhD, of the University of British Columbia, to develop therapeutic antibodies for the treatment of Duchenne muscular dystrophy-associated fibrosis. “It is immensely satisfying to see many years of investment in the basic science now being translated towards therapies for patients in need, and […]