Jesse's Journey Foundation

Defeat Duchenne. Change Lives.

Research Updates

EIM Could Be Reliable Method to Assess Effects of Therapy in DMD Trials

A technique called electrical impedance myography (EIM) can help detect muscle deterioration in people with Duchenne muscular dystrophy (DMD), according to a study published in the scientific journal Annals of Neurology. The method is also able to identify the therapeutic effects of corticosteroids and could therefore be a good biomarker in clinical trials. To evaluate […]


Translarna in Canada

Translarna received marketing authorization by the European Medicines Agency and is available in Europe and regions that reference that authorization. Currently, Translarna has not received marketing authorization in Canada. Based on the outcome of ongoing regulatory interactions we are developing a plan to file a New Drug Submission for Translarna in 2017. PTC remains committed […]


Scientists replace piece of gene mutated in Duchenne muscular dystrophy in effort to make healthy

AUGUSTA, Ga. (Dec. 19, 2016) – Scientists are using “gene scissors” to cut off the code of a defective gene that results in progressively weaker muscles and death in Duchenne muscular dystrophy and replace it with a synthetic code they hope will one day restore healthy life to these patients. “We want to use genetically […]


Catabasis Pharmaceuticals Provides Edasalonexent and Rare Disease Pipeline Updates at Investor Day

CAMBRIDGE, Mass.–(BUSINESS WIRE)–Catabasis Pharmaceuticals, Inc. (NASDAQ:CATB), a clinical-stage biopharmaceutical company, today is holding its first Investor Day and will provide an in-depth review of the Company’s strategy and pipeline in rare diseases, including edasalonexent (CAT-1004) and other programs. Guest speakers will include Craig McDonald, M.D., UC Davis NeuroNEXT Program Director, University of California, and H. […]


New research increases understanding of Duchenne muscular dystrophy

BINGHAMTON, NY – A new paper, co-written by faculty at Binghamton University, State University of New York, increases the understanding of Duchenne muscular dystrophy (DMD)–one of the most common lethal genetic disorders–and points to potential therapeutic approaches. “The findings suggest that the immune system has an important role in the muscle disease of Duchenne muscular […]


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 […]