Jesse's Journey Foundation

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Research Updates

Sarepta Therapeutics: Reports Positive Clinical Results from Phase 2 MOMENTUM Study of SRP-5051

Sarepta Therapeutics has announced encouraging results on their PPMO drug candidate for individuals with Duchenne muscular dystrophy amenable to Exon 51 skipping. Preliminary data for the 30mg/kg dose shows 6.55% mean dystrophin expression and predicting longer-term use will achieve greater than 10% dystrophin production. Hypomagnesemia was identified as a safety concern, but all cases are [...]

Pfizer: Canada Launches First Gene Therapy Trial for Duchenne

Ground-breaking news, Canada has launched the first gene therapy trial for Duchenne muscular dystrophy. Pfizer’s investigational gene therapy treatment is now recruiting in Canada at Children's Hospital - London Health Sciences Centre in London, Ontario. This is an international study and their first site to open in North America. Boys with a confirmed diagnosis of [...]

FibroGen: Receives Fast Track Designation from FDA for Pamrevlumab

FibroGen has received Fast Track designation from the U.S. Food and Drug Administration (FDA) for pamrevlumab to treat muscle fibrosis in Duchenne muscular dystrophy. This designation follows a review of the Phase 2 clinical data from a single-arm trial in non-ambulatory patients with Duchenne. What is muscle fibrosis? Muscle fibrosis is the formation of excess […]

Muscular Dystrophy Association (MDA): 2021 Conference Update

Jesse’s Journey is pleased to share three treatment updates for Duchenne muscular dystrophy as presented at the recent Muscular Dystrophy Association (MDA) Virtual Clinical & Scientific Conference. Pfizer: PF-06939926 The ongoing Phase 1b study (NCT03362502) is evaluating the safety and tolerability of PF-06939926 in boys who can walk at least 10 meters (about 33 feet) [...]

Solid Biosciences: IGNITE DMD Clinical Trial Update

Solid Biosciences has provided an update on the safety and efficacy data from their IGNITE DMD Phase I/II clinical trial: We are pleased to share an update regarding the safety and efficacy data from our ongoing IGNITE DMD phase I/II clinical trial, as announced in the press release issued this afternoon. Additionally, we are excited to announce […]

NS Pharma, Inc.: RACER53 Recruitment

The Hospital for Sick Children (SickKids) in Toronto is currently recruiting boys with Duchenne muscular dystrophy aged 4-7 with mutations amenable to exon 53 skipping for the NS Pharma, Inc. RACER53 clinical trial investigating the new drug, viltolarsen. Click here to learn more and find out if you might qualify for this study: RACER53 Study Overview […]

VISION-DMD: White Paper on Return of Clinical Trial Data

VISION-DMD published a white paper on how to return individual clinical trial results back to patients. It focuses on addressing the ethical and technical challenges, using the vamorolone clinical trial experience. In short: Often, individual patient data is not returned to the participants, even when this is requested. ReveraGen received a grant to develop an [...]

Santhera Pharmaceuticals: VISION-DMD Clinical Trial Update

Santhera Pharmaceuticals has announced the completion of the first 6-month period of  VISION-DMD, a clinical trial investigating vamorolone. If there are positive top-line results in the data, to be released sometime in the second quarter of this year, it could lead to a submission to the Food and Drug Administration (FDA) in the first quarter [...]