Jesse's Journey Foundation

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Research Updates

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 […]