Jesse's Journey Foundation

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Research Updates

Sarepta Bolstered by Doctor Letter Urging FDA Approval of DMD Drug

Thirty-six doctors, all experts in Duchenne muscular dystrophy, or DMD, have written a letter to the U.S. Food and Drug Administration urging the agency to approve Sarepta Therapeutics’ (SRPT – Get Report) experimental therapy eteplirsen. The lobbying effort seeking to bolster support for eteplirsen and Sarepta — and counter previously released FDA criticism of the drug — […]


Summit Therapeutics’ study identifies DMD biomarkers

Summit Therapeutics plc (NASDAQ: SMMT LON:SUMM) has identified biomarkers to relate muscle regeneration and the presence of utrophin protein in patients with Duchenne (DMD) and Becker muscular dystrophy (BMD). DMD is caused by the absence of dystrophin protein and it is believed utrophin modulation, a naturally occurring protein similar to dystrophin, could slow or even stop the […]


PTC Therapeutics Provides Update on Health Canada Review of Translarna™ (ataluren) for the Treatment of Duchenne Muscular Dystrophy

SOUTH PLAINFIELD, N.J., March 14, 2016 /PRNewswire/ — PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced that the company intends to submit the results of the recently completed Phase 3 ACT DMD study for review by Health Canada as part of the New Drug Submission (NDS) for Translarna™ (ataluren) to treat nonsense mutation Duchenne muscular dystrophy (nmDMD).  In order to […]


Federal Government Stem Cell Research Funding Good News: Jesse’s Journey

Jesse’s Journey is applauding Wednesday’s announcement the federal government will help fund a stem cell therapy development facility in Toronto. Prime Minister Justin Trudeau said the government will provide $20 million to the Centre for Commercialization of Regenerative Medicine. Trudeau said supporting the facility will have significant benefits for innovative, health-related technology in Canada and […]


BioMarin Fails to Win Approval for Drug to Treat Duchenne

BioMarin Pharmaceutical Inc. failed to win U.S. approval for a drug to treat Duchenne muscular dystrophy, a deadly genetic disease that usually affects young boys. The Food and Drug Administration rejected the once-a-week injection known as Kyndrisa, according to a statement from the company Thursday. A panel of outside advisers voted in November that the medication, known chemically […]


Trudeau announces $20-million in funding for Toronto stem cell research

The federal government will provide $20 million to the Centre for Commercialization of Regenerative Medicine to help establish a stem-cell therapy development facility in Toronto. The Centre for Advanced Therapeutic Cell Technologies will be the first such facility in the world to use a collaborative approach between research institutions and industry to solve cell therapy […]


PTC Completes Rolling NDA Submission to FDA and Submits Phase 3 ACT DMD Clinical Trial Results to EMA for Translarna™ (ataluren) for Treatment of Nonsense Mutation Duchenne Muscular Dystrophy

SOUTH PLAINFIELD, N.J., Jan. 8, 2016 /PRNewswire/ — PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced the completion of its rolling submission of a New Drug Application (NDA) to the United States Food and Drug Administration (FDA) for Translarna™ (ataluren), an oral, first-in-class, protein restoration therapy for the treatment of nonsense mutation Duchenne muscular dystrophy (nmDMD).  PTC has also […]


Cancer drug shows promise for treating Duchenne muscular dystrophy

A drug commonly used to treat leukaemia is showing potential as a treatment that could slow the progression of the muscle-wasting condition, Duchenne muscular dystrophy. Duchenne muscular dystrophy most commonly affects boys, with around 2,400 people in the UK affected by the condition. There is currently no cure and most patients are not expected to […]


Gene-editing technique successfully stops progression of Duchenne muscular dystrophy

DALLAS – Dec. 31, 2015 – Using a new gene-editing technique, a team of scientists from UT Southwestern Medical Center stopped progression of Duchenne muscular dystrophy (DMD) in young mice. If efficiently and safely scaled up in DMD patients, this technique could lead to one of the first successful genome editing-based treatments for this fatal […]