Jesse's Journey Foundation

— 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 […]

PTC Therapeutics has announced three Canadian sites for their clinical trial known as Study 041 for Duchenne muscular dystrophy.  Study 041 is looking at how the investigational treatment, ataluren, affects ability to walk and endurance in males aged five years and older with nmDMD, compared to placebo. More information about the study can be found by clicking here […]

The first patient has been enrolled in a Phase 2 trial evaluating the effectiveness and safety of MNK-1411, an investigational therapy for Duchenne muscular dystrophy (DMD), announced Mallinckrodt Pharmaceuticals, the treatment’s developer. The company also reported that MNK-1411 was granted orphan medicinal product status by the European Medicines Agency, facilitating the development and eventually the approval process for the treatment candidate. The therapy was given orphan drug designation in […]

Gavin Ward, a spunky 8-year-old Arkansas boy with Duchenne, enjoys building contraptions with his Lego set and watching video games. He also walks up to 18,000 steps a day and accompanies his father, Bruce, on outings near their home in Royal, about 70 miles west of Little Rock. But what makes Gavin stand out in […]

SAN ANTONIO, TEXAS, Aug. 1, 2018 — A protein known to drive nerve cell survival in the brain and spinal cord might also protect failing hearts in children and young adults with Duchenne muscular dystrophy, according to preliminary research presented at the American Heart Association’s Basic Cardiovascular Sciences Scientific Sessions, a premier global exchange of […]

A Phase 1/2 trial evaluating Sarepta’s microdystrophin gene therapy candidate for Duchenne muscular dystrophy (DMD) was placed on clinical hold by the U.S. Food and Drug Administration. The FDA found trace amounts of plasmid DNA in the raw material used to make the drug. The fragments don’t seem harmful in preliminary tests and the dosing of patients by the end of […]

Insilico Medicine and A2A Pharmaceuticals, two leading artificial intelligence (AI) biotechs, are collaborating to create a new company called Consortium.AI, which will advance AI for the development of new molecules to treat Duchenne muscular dystrophy (DMD) and other orphan diseases. Consortium.AI will feature computational new candidates designed to target specific diseases that were validated through Insilico’s AI systems. A2A Pharmaceuticals […]