Jesse's Journey Foundation

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Research Updates

Research Grant Recipient: Satellos Bioscience

Jesse's Journey is proud to announce a research partnership and infrastructure grant to support the development of Satellos' novel approach to treating Duchenne. Satellos scientists are developing small molecule drugs that they believe will restore faulty regeneration and repair observed in the muscles of patients with Duchenne and potentially other degenerative muscle disorders. The company's drug [...]

NS Pharma: VILTEPSO® (viltolarsen) Injection Long-Term Efficacy and Safety Data

N.S. Pharma, Inc. has announced new long-term efficacy and safety data from the open-label extension trial of a Phase 2 study of VILTEPSO® (viltolarsen) injection. VILTEPSO is an exon-skipping therapy for individuals with Duchenne muscular dystrophy with deletion mutations amenable to exon 53 skipping. The efficacy and safety results were based on interim analyses at week [...]

Italfarmaco: Topline Data from Phase 2 Trial with Givinostat

Italfarmaco has announced results for their Givinostat trial in Becker muscle dystrophy (BMD). Although the change in total fibrosis in muscle biopsy did not show a significant difference in 51 adult males with BMD, it did show a significant difference from placebo on muscle MRI confirming the ability of Givinostat to counteract muscle deterioration. Italfarmaco [...]

Jesse’s Journey and PPPMD Award Clinical Fellowship in Duchenne Endocrinology and Bone Fragility

Jesse's Journey and Parent Project Muscular Dystrophy Award $172,000 (CAD) Clinical Fellowship in Duchenne Endocrinology and Bone Fragility. Award Will Sponsor a Two-Year Fellowship Under Dr. Leanne Ward, the University of Ottawa with Focus on Endocrine and Bone Complications of Duchenne. Parent Project Muscular Dystrophy (PPMD), a US nonprofit organization leading the fight to end [...]

Santhera and ReveraGen: Positive Topline Results with Vamorolone in VISION-DMD Study

Santhera and ReveraGen have announced positive topline results for vamorolone, a steroid alternative for individuals living with Duchenne muscular dystrophy (DMD). Their pivotal VISION-DMD study in boys 4 to <7 years old demonstrated benefits across multiply timed tests compared to placebo (boys not on treatment) and similar benefits to individuals taking prednisone. Vamorolone also showed [...]

Solid Biosciences: IGNITE DMD Phase I/II Clinical Trial Update

Jesse’s Journey is pleased to share the letter for the Duchenne community from Solid Biosciences, providing a business update and including a dosing update in the IGNITE DMD Phase I/II clinical trial and promising long-term biopsy data from prior patients dosed with SGT-001 in the high dose cohort at 2E14 vg/kg. They also announced the [...]

Sarepta Therapeutics: SRP-9001 (ENDEAVOR) Gene Therapy Study Update

Jesse’s Journey is pleased to share an update from Sarepta Therapeutics on their Gene Therapy for DMD SRP-9001. The results from the first 11 patients enrolled showed an increase in micro-dystrophin expression and a consistent safety profile. “We are delighted by these seminal results from the ENDEAVOR Study, our first trial results with SRP-9001 made [...]

Capricor Therapeutics: Expands its Exosome Platform Technology Portfolio

Capricor Therapeutics has announced that it has signed an exclusive, worldwide licensing agreement with Johns Hopkins University (JHU) to include engineered exosomes for vaccines and therapeutics as part of its exosome technology portfolio. Exosome-based therapy is an exciting area for Duchenne muscular dystrophy. We hope it will become the next generation of gene therapy without [...]

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 [...]