Jesse's Journey Foundation

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News & Resources

Sarepta: Launch of EMBARK – Gene Therapy Study

Jesse’s Journey is pleased to share with our Canadian Duchenne community the latest release from Sarepta Therapeutics. They recently announced the initiation of EMBARK and the advancement of the SRP-9001 program. This represents the first pivotal double-blind gene therapy trial in Duchenne, initiated in the US, Europe and Asia. Doug Ingram, CEO and president, said: [...]

Pfizer: Change in Gene Therapy Protocol

Pfizer has announced a change to its gene therapy clinical program. Pfizer’s investigational gene therapy trial started recruitment in Canada in March 2021. They recently announced they will be changing their trial protocol to exclude patients with any mutation (exon deletion, duplication, insertion, or point mutation) affecting exons 9-13, or deletions that affect both exon [...]

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 [...]

Health Canada: National Strategy for Drugs for Rare Diseases Update

In January 2021, Health Canada launched a national online initiative to engage Canadians in the development of a National Strategy for Drugs for Rare Diseases. More than 650 patients, families, stakeholders, and patient organizations - like Jesse's Journey - provided their diverse perspectives on getting better access to the effective drugs they need. Throughout the [...]

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 [...]

Longtime Volunteer Walks Across Canada

This Canada Day, long-time Jesse's Journey volunteer Harry Norris will complete his goal of walking across Canada - without leaving his hometown of Mitchell, Ontario.  Beginning in March 2020, the 76-year-old retired school principal started a journey that saw him walk more than 8,200 kilometres - a distance that would take him coast to coast.  [...]

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 [...]

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