Jesse's Journey Foundation

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News & Resources

Update from PTC Therapeutics

Dear Duchenne Community, As we enter the holiday season, I wanted to thank the community for all your support throughout the regulatory process for Translarna™ (ataluren) with the FDA. Your voice as passionate advocates has been instrumental in this process. As you know, we filed a Formal Dispute Resolution Request (FDRR) with the FDA a […]

Duchenne Research: 2017 in Review

2017 has been an exciting year for Duchenne muscular dystrophy research! As we get close to the end of the year, we’re looking back at some of the stories that caught our attention. You can find these and other news stories here. Jesse’s Journey grants more than $1 million internationally to Duchenne researchers In May, […]

Capricor Set to Launch Phase 2 Trial of Cell Therapy CAP-1002 in Advanced DMD Patients

Capricor Therapeutics has received a green light from the U.S. Food and Drug Administration to launch a Phase 2 study of CAP-1002 cell therapy in children and adults with advanced Duchenne muscular dystrophy. Capricor expects to open the trial to patient enrollment in early 2018 and, if successful, to begin steps needed to receive FDA approval of CAP-1002 […]

Solid Biosciences Readies Clinical Trial of SGT-001, a Potential Duchenne MD Gene Therapy

Solid Biosciences announced the pending start of a first clinical trial to test the company’s leading investigational therapy – called SGT-001, a microdystrophin gene transfer candidate, as a potential treatment for Duchenne muscular dystrophy (DMD). The Phase 1/2 trial, called IGNITE DMD, will soon open for patient enrollment in the United States. “For more than three […]

#ThankYouThursday – 20 years of progress

On November 28, we celebrated Giving Tuesday – a day when people come together to celebrate philanthropy and support their favourite causes. Today, it’s Thank You Thursday. Today, we’re saying Thank You to everyone who has supported Jesse’s Journey for more than 20 years. It’s amazing to look back and see how far we have […]

Sarepta Therapeutics and Nationwide Children’s Hospital Announce FDA Clearance of IND for Micro-Dystrophin Gene Therapy Program for the Treatment of Duchenne Muscular Dystrophy

CAMBRIDGE, Mass., Nov. 06, 2017 (GLOBE NEWSWIRE) — Sarepta Therapeutics, Inc. (NASDAQ:SRPT), a commercial-stage biopharmaceutical company focused on the discovery and development of precision genetic medicines to treat rare neuromuscular diseases, today announced that Nationwide Children’s Hospital has received clearance from the U.S. Food and Drug Administration (FDA) for its Investigational New Drug (IND) application […]

A Letter from PTC Therapeutics

Dear Duchenne Community, As you might have seen, the Office of Drug Evaluation I of the FDA provided a Complete Response Letter (CRL) for the application to market ataluren in the United States. This means that the FDA believes ataluren cannot be approved at this time. We are extremely disappointed and we strongly disagree with […]

FDA Rejects New Drug Application for Translarna to Treat DMD

The U.S. Food and Drug Administration decided not to approve Translarna (ataluren), by PTC Therapeutics, as a treatment for specific types of Duchenne muscular dystrophy caused by “nonsense” mutations. Although not entirely unexpected, the decision was disappointing to many in the Duchenne community. On Sept. 28, an FDA advisory panel concluded that evidence to support Translarna’s effectiveness as a therapy […]

Jesse’s Journey Has Lost a Dear Friend

Angie Stewart  September 1 1962 – October 18 2017 Jesse’s Journey has lost a dear friend. Angie Stewart was the wife of Ron (Sam) and mother of 2 boys: Steven (who had Duchenne muscular dystrophy and passed away in 2009) and Matthew. Angie was a beautiful person and an inspiration to everyone she met. Her […]

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