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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 […]


Treating Duchenne muscular dystrophy focus of new research partnership

Toronto, Ontario – Muscular Dystrophy Canada (MDC) and The Foundation for Gene & Cell Therapy (Jesse’s Journey) are joining forces to accelerate ground-breaking research focused on new treatments for Duchenne muscular dystrophy to the sum of $600,000. Through this partnership, MDC will provide $300,000 with Jesse’s Journey matching the commitment. Two research projects will be […]


International Preferences for Duchenne Treatments Study

The International Preferences for Duchenne Treatment Study is now open in Canada. Jesse’s Journey has partnered with Johns Hopkins University, Parent Project Muscular Dystrophy, and other Duchenne advocacy organizations from around the world on this important study. This survey is the first international study to measure the preferences and priorities of people with Duchenne and […]


Jesse’s Journey Grants 10 Millionth Dollar to Research

On November 21, Jesse’s Journey announced to donors, volunteers, and families who are facing Duchenne muscular dystrophy that as of 2018, Jesse’s Journey has granted a cumulative total of more than $10 million to research. For more than 20 years, Jesse’s Journey has been dedicated to funding research focused on Duchenne muscular dystrophy – the […]


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 […]


Top 10 Muscular Dystrophy Articles of 2018

Throughout 2018, Muscular Dystrophy News Today provided daily coverage of new therapeutic strategies, clinical trials, and other topics related to muscular dystrophy (MD). As we look forward to reporting more news to patients, family members, and caregivers dealing with MD in 2019, here are the Top 10 most-read articles of 2018, with a brief description of their relevance to the […]


New drug for Duchenne muscular dystrophy clears phase 1 clinical trial testing in boys

Amsterdam, February 21, 2019 – Patients with Duchenne muscular dystrophy (DMD) have few treatment options. Medications currently available or in development either target only a subset of DMD patients with a particular genetic mutation or cause significant side effects. The investigational drug edasalonexent, an oral NF-κB inhibitor, has the potential to slow the progression of […]


Gene Therapy SGT-001 Shows Signs of Microdystrophin Production in Muscles of Boys in IGNITE DMD Trial

Preliminary results of a Phase 1/2 study into the gene therapy candidate SGT-001 in Duchenne muscular dystrophy (DMD) patients show low levels of microdystrophin in patients’ muscles after three months of treatment with an initial low dose and continued safety, Solid Bioscience, the therapy’s developer, reported. Continued safety was also evident, the company said in a press release. It added […]


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