Jesse's Journey Foundation

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News & Resources

Sarepta’s Duchenne MD trial back on schedule

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 […]

Pull a Plane to Defeat Duchenne

On Saturday, September 22 more than 100 people came out to the Jet Aircraft Museum in London, Ontario for the first “Pull a Plane to Defeat Duchenne”. This first-time event was held in partnership with Western University’s Commercial Aviation Management program. Participants teamed up to pull a 10,000lb, beautifully-restored Canadian T33 jet 50 metres across the […]

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 […]

Jesse’s Journey Welcomes New Executive Director

We have a new leader of the team here at Jesse’s Journey.   We are pleased to announce that Perry Esler will be assuming the role of Executive Director on October 1st. Perry is no stranger to Jesse’s Journey, having been on the road with us in 1995. He comes to us with more than […]

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 […]

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