Jesse's Journey Foundation

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Research Updates

Potential DMD Therapy Vamorolone Shows Positive Effects in Phase 2a Trial, Study Reports

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 […]


Sarepta Receives Negative CHMP Re-examination Opinion for Eteplirsen

— 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 […]


Canadian Collaboration Gives Funding Boost to Duchenne MD Research

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 Study 041 Canadian trial sites

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 […]


First Patient Enrolled in Phase 2 Trial of MNK-1411, Mallinckrodt’s Investigational Therapy for DMD

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 […]


A protein could be key to preserving heart function in Duchenne muscular dystrophy

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 […]


FDA Places Hold on Phase 1/2 Trial of Sarepta’s Gene Therapy for DMD

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 […]