Jesse's Journey Foundation

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Research Updates

PTC Therapeutics Announces First-Ever Winners of Its PRIORITY Program To Support Research Projects In Rare, Genetic Disorders

– $545,000 awarded to help improve Duchenne muscular dystrophy diagnosis and screening across the globe –SOUTH PLAINFIELD, N.J., May 20, 2019 /PRNewswire/ — PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced the three funding recipients from PRIORITY, an annual program to support outstanding clinical research by the rare genetic disorders community. This year’s funding will accelerate innovative research projects to improve Duchenne […]


Helping Canadians get faster access to the prescription drugs they need to live healthier lives

Government of Canada consults on new approach to fast-track the review of treatments for serious, rare and debilitating diseases OTTAWA, May 13, 2019 /CNW/ – The Government of Canada is committed to helping all Canadians access affordable treatment options they need to help them improve and maintain their health. For many Canadians, managing the symptoms of rare or debilitating diseases can be […]


EGF Protein Preserves Muscle Strength, Promotes Muscle Regeneration in DMD Mice, Study Shows

A protein called epidermal growth factor (EGF) can help preserve muscle strength and increase muscle regeneration in a mouse model of Duchenne muscular dystrophy (DMD), a finding that may pave the way for new treatment strategies for DMD, researchers said. The study, “EGFR-Aurka Signaling Rescues Polarity and Regeneration Defects in Dystrophin-Deficient Muscle Stem Cells by Increasing Asymmetric Divisions,” […]


Translarna™ (ataluren) is the First Therapy Approved in Brazil for Duchenne Muscular Dystrophy

– Ambulatory Duchenne patients who are 5 years and older with a nonsense mutation can now access a treatment that targets the underlying cause – SOUTH PLAINFIELD, N.J., April 29, 2019 /PRNewswire/ — PTC Therapeutics, Inc. today announced that Translarna™ (ataluren) has been granted marketing approval from the Brazilian National Health Surveillance Agency (ANVISA) under rare diseases […]


Raising Both Dystrophin and Utrophin Levels May Rescue Muscles in DMD, Mouse Study Suggests

Using a genetic engineering tool to restore dystrophin while raising levels of utrophin, a similar protein, leads to better improvements in muscle function than either approach alone, research in a mouse model of Duchenne muscular dystrophy (DMD) reports. The study, “The potential of utrophin and dystrophin combination therapies for Duchenne muscular dystrophy,” appeared in the journal Human Molecular […]


DMD Gene Therapy Showing ‘Very Encouraging’ Results at 9 Months in Phase 1/2 Study, Sarepta Reports

New nine-month data on four boys with Duchenne muscular dystrophy (DMD) enrolled in Study-101 testing Sarepta Therapeutics‘ micro-dystrophin gene therapy continues to show “very encouraging” results, company officials said. These updated data, given in a recent investors’ webinar, show the gene therapy resulted in 81.2% increase in dystrophin protein expression in the muscles, accompanied by marked improvements in boys’ […]


CRISPR-Cas9 corrects Duchenne muscular dystrophy exon 44 deletion mutations in mice and human cells

Abstract Mutations in the dystrophin gene cause Duchenne muscular dystrophy (DMD), which is characterized by lethal degeneration of cardiac and skeletal muscles. Mutations that delete exon 44 of the dystrophin gene represent one of the most common causes of DMD and can be corrected in ~12% of patients by editing surrounding exons, which restores the […]


Edasalonexent May Limit Disease Progression in Boys with Duchenne MD, Phase 1/2 Trial Shows

The investigational treatment edasalonexent was well-tolerated, led to no safety issues, and blocked a crucial muscle disease protein in boys with Duchenne muscular dystrophy(DMD), according to part 1 results of a Phase 1/2 trial. The study, “Phase 1 Study of Edasalonexent (CAT-1004), an Oral NF-κB Inhibitor, in Pediatric Patients with Duchenne Muscular Dystrophy,” was published in the Journal of […]