Jesse's Journey Foundation

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News & Resources

#AAN2018 — Exondys 51 Found to Be Safe in Young Infant with DMD, Preliminary Data Shows

Treatment with Exondys 51 (eteplirsen) was found to be safe and well-tolerated in a very young infant with Duchenne muscular dystrophy (DMD), preliminary data of a follow-up study shows. Initial results of the study will be the subject of a poster presentation at the 2018 Annual Meeting of the American Academy of Neurology (AAN), taking place April 21-27 in Los Angeles. The presentation […]


National Volunteer Week

It’s National Volunteer Week in Canada and we want to take a moment to thank all of the incredible volunteers who generously give their time to Jesse’s Journey. When John Davidson walked across Canada in 1998, volunteers played a crucial role in keeping him moving. From coast to coast, so many dedicated people took time […]


NIH Agency Pioneers Collaborative Research into Rare Diseases

A little-known government entity within the National Institutes of Health (NIH) is helping to lead U.S. efforts to speed up the development of therapies for some 7,000 rare diseases. The Office of Rare Diseases Research (ORDR), headquartered in Bethesda, Maryland, was established in 1993 within the NIH Office of the Director. In 2011, it became part of the NIH’s National […]


Pfizer launches Duchenne gene therapy study, joining Sarepta and Solid

Pfizer has officially entered the race to cure Duchenne muscular dystrophy with gene therapy, joining Cambridge-based Sarepta Therapeutics and Solid Biosciences as the only companies to use the experimental technology on a boy with the muscle-wasting disease. Pfizer (NYSE: PFE) announced on Thursday that the first patient in a small, early-stage trial of its gene therapy drug had […]


A Note From John Davidson, April 10

As I promised you, this is the first of the weekly postings as I look back at the walk across Canada that began 20 years ago today. So here we go! This is it. Day number one in St. John’s, Newfoundland. After seven months of training, today the distance really starts to mean something. Looking [...]

Duchenne Muscular Dystrophy: As Survival Increases, New Focus on Quality of Life

David Birnkrant, MD Professor of Pediatrics, Case Western Reserve University School of Medicine Director of Pediatric Pulmonology & Student Education, MetroHealth Medical Center MedicalResearch.com: What is the background for this study? What are the main findings? Response: This study updates guidance on all aspects of the multi-disciplinary care of patients with Duchenne muscular dystrophy (DMD). The […]


DMD Trial of Dystrophin Gene Therapy SGT-001 Put on Clinical Hold by FDA

The U.S. Food and Drug Administration has placed a clinical hold on a Phase 1/2 trial testing SGT-001, a dystrophin gene therapy intended to treat Duchenne muscular dystrophy (DMD), after the first patient dosed was hospitalized with a possibly serious reaction. Solid Biosciences, the therapy’s developer, announced that within days of being given SGT-001 at 5E13 vg/kg […]


Approval Received for Phase II Trial of ATL1102 in Duchenne Muscular Dystrophy

MELBOURNE, Australia, February 28, 2018 (Newswire.com) – Antisense Therapeutics (“ANP” or the “Company”) is pleased to advise that the Company has received approval from the Royal Children’s Hospital (RCH), Melbourne Human Research Ethics Committee, to undertake a Phase II clinical trial of its immunomodulatory therapy, ATL1102, in patients with Duchenne Muscular Dystrophy (DMD), an incurable muscle-wasting […]


New Published Study Reports Cardiosphere-Derived Cells Improved Skeletal and Cardiac Muscle Function in Mouse Model of Duchenne Muscular Dystrophy

Cedars-Sinai Researchers Say Findings Lay Groundwork for Testing of CAP-1002 in Patients with Duchenne Muscular Dystrophy LOS ANGELES, Feb. 22, 2018 (GLOBE NEWSWIRE) — Capricor Therapeutics (NASDAQ:CAPR) today announced the online publication in Stem Cell Reports of a new study by researchers at the Smidt Heart Institute at Cedars-Sinai Medical Center, who found that cardiosphere-derived cells (CDCs) improved cardiac […]


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