Jesse's Journey Foundation

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Research Updates

FDA grants accelerated approval to first drug for Duchenne muscular dystrophy

The U.S. Food and Drug Administration today approved Exondys 51 (eteplirsen) injection, the first drug approved to treat patients with Duchenne muscular dystrophy (DMD). Exondys 51 is specifically indicated for patients who have a confirmed mutation of the dystrophin gene amenable to exon 51 skipping, which affects about 13 percent of the population with DMD. […]


NHS England Enables Access to Translarna™ (ataluren) For Patients with Nonsense Mutation Duchenne Muscular Dystrophy

–Important decision allows reimbursed access to Translarna, the first approved therapy to treat the underlying cause of Duchenne muscular dystrophy– SOUTH PLAINFIELD, N.J., July 7, 2016 /PRNewswire/ — PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced that the company and NHS England have successfully negotiated a Managed Access Agreement (MAA) for Translarna (ataluren) for ambulatory patients […]


SickKids’ new Paediatrician-in-Chief weighs in on how far we’ve come and the power of integration in genetics

On July 1, Dr. Ronald (Ronni) Cohn stepped into his new role as Paediatrician-in-Chief at The Hospital for Sick Children (SickKids). As Cohn, Senior Scientist in Genetics & Genome Biology, prepares to lead SickKids into a genetics-fuelled future, he reflects on how the field has evolved, and looks ahead at the potential of individualized medicine […]


PTC Initiates Phase 2 Clinical Trial of Translarna™ (ataluren) for Treatment of Nonsense Mutation Duchenne Muscular Dystrophy in Pediatric Patients Between the Ages of Two and Five Years

PTC Therapeutics has announced that they have initiated a Phase 2 clinical trial of Translarna™ (ataluren) for children 2 to 5 years old. Details of site locations, etc. are on clinicaltrials.gov.  Below is the announcement from PTC. We are pleased to inform you that PTC Therapeutics has initiated a Phase 2 clinical trial of Translarna™ (ataluren), […]


Marathon Pharmaceuticals submits deflazacort NDA to treat Duchenne muscular dystrophy

Marathon Pharmaceuticals has submitted a new drug application (NDA) to the US Food and Drug Administration (FDA), seeking approval for its deflazacort investigational drug to treat Duchenne muscular dystrophy (DMD). Deflazacort is a glucocorticoid with anti-inflammatory and immunosuppressant properties. It has fast track status, orphan drug designation and rare pediatric disease designation for DMD. Click […]


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 […]