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News & Resources

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

May 20, 2021

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 [...]
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World Duchenne Awareness Day: September 7, 2021

May 13, 2021

September 7 is World Duchenne Awareness Day. On this day, Jesse's Journey joins forces with organizations from around the world to raise awareness for Duchenne and Becker Muscular Dystrophy (DMD/BMD). Every year, the World Duchenne Organization sets a special theme to the day that that deserves more attention. This year it will be ‘Adult Life [...]
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Capricor Therapeutics: Expands its Exosome Platform Technology Portfolio

May 4, 2021

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

May 4, 2021

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 [...]
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Pfizer: Canada Launches First Gene Therapy Trial for Duchenne

May 4, 2021

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 [...]
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Jesse’s Journey: Grants $1.7 million to Duchenne muscular dystrophy research

May 4, 2021

Jesse's Journey, Canada's leader in Duchenne muscular dystrophy funded research, is proud to announce that it will grant $1.7M toward four promising research projects this year - the largest annual amount in its 26-year history. Duchenne muscular dystrophy is a life-threatening, progressive, neuromuscular disorder with no cure. Affecting approximately 1 in every 5,000 boys in [...]
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Open Letter to Minister: Reduce the Timing Between Doses for Patients with Neuromuscular Diseases

April 29, 2021

The Honourable Patty Hajdu, P.C., M.P. Minister of Health Via email: [email protected] Dear Minister Hajdu: We are a group of physicians, health care workers and stakeholders who care for patients with neuromuscular diseases across Canada. We are writing this open letter to request for patients with neuromuscular diseases to be eligible for vaccination doses earlier [...]
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FibroGen: Receives Fast Track Designation from FDA for Pamrevlumab

April 13, 2021

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 […]

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Health Canada: Recommendations for National Rare Disease Strategy

March 30, 2021

Over the last month, Health Canada has opened up a consultation for organizations like Jesse's Journey, to provide recommendations on a discussion paper outlining ways to address access to high-cost rare disease drugs in Canada. There were three different ways to respond: Questionnaire Participate in a public town hall Written submission Jesse's Journey submitted input [...]
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