Jesse's Journey Foundation

Defeat Duchenne. Change Lives.

Research Updates

Translarna not recommended in Scotland

Translarna, the first ever treatment to address the underlying causes of Duchenne muscular dystrophy, has received a negative funding recommendation from the Scottish Medicines Consortium (SMC) on the basis of cost. This is despite the proactivity and efforts of Action Duchenne, families, and other charities to inform SMCs evaluation and ensure that the patient voice remained at […]


Defazacort Improves Lung Function in Advanced Duchenne MD Patients, Marathon Reports

Duchenne muscular dystrophy (DMD) patients at more advanced disease stages, those reliant on wheelchairs, showed improved lung function when treated with deflazacort compared to prednisone, Marathon Pharmaceuticals reported in a presentation at the recent MDA Clinical Conference in Arlington, Virginia. The presentation, “Effect of deflazacort and prednisone versus placebo on pulmonary function in boys with Duchenne Muscular Dystrophy who have lost ambulation,” added to […]


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 […]