Jesse's Journey Foundation

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News & Resources

Sarepta Therapeutics: Top-Line Results for Part 1 of Study 102 SRP-9001 Gene Therapy

On January 7, Sarepta Therapeutics announced their topline results from their gene therapy candidate SRP-9001. This study is an ongoing, randomized, double-blinded, placebo-controlled trial evaluating the safety, efficacy and tolerability of a single dose of SRP-9001 in 41 Duchenne patients aged 4-7. The study met its biological primary endpoint of micro-dystrophin expression at 12 weeks. […]

2020 Year in Review

While we look ahead with hope, possibility, and vision, we take time to reflect on a year like no other. We started January 2020 with fresh excitement to celebrate our 25th anniversary and share a new three-year strategic plan focussed on engaging Duchenne families across Canada like never before. We are proud of what we [...]

2021: New Year Filled with Hope

We're starting 2021 filled with gratitude. Throughout November and December, you picked up the pieces to the complex puzzle of Duchenne muscular dystrophy and helped create the beautiful mosaic of HOPE. With your generosity, we surpassed our goal of $100,000 to invest in research that will allow boys and young men around the world to live longer, healthier lives. This would not [...]

COVID-19 and Duchenne

With the growing concerns regarding COVID-19 (“coronavirus”) across Canadian communities and the world, our hearts go out to all those who've been impacted. We also want to show our profound gratitude to the health care professionals and everyone on the front lines of this pandemic. Jesse's Journey is here to support the Duchenne community during [...]

Sarepta Therapeutics: Positive Clinical Results from MOMENTUM – Phase 2 Clinical Trial of SRP-5051

Sarepta has announced encouraging interim results with their SRP-5051 exon skipping molecule for both ambulatory (walking) and non-ambulatory (non-walking) Duchenne patients amenable to exon 51 skipping ages 4-21 years. This new second-generation exon skipping molecule in early clinical trials shows higher tissue exposure than Eteplirsen, with a favourable safety profile. They continue to test higher […]

ReveraGen: Receives $3.3 Million from National Institutes of Health (NIH)

ReveraGen BioPharma, the developer of vamorolone, received a 3.3 million dollar grant from the National Institutes of Health (NIH) to support the commercialization of this safer alternative for corticosteroids treatment. This will advance vamorolone towards FDA submission and commercialization in the USA. This is excellent news for the Duchenne community and is still pending their […]

Sarepta Therapeutics: SRP-9001 Gene Therapy Update

About SRP-9001 (AAVrh74.MHCK7.micro-dystrophin) SRP-9001 is an investigational gene transfer therapy intended to deliver the micro-dystrophin-encoding gene to muscle tissue for the targeted production of the micro-dystrophin protein. Sarepta is responsible for global development and manufacturing of SRP-9001 and plans to commercialize SRP-9001 in the United States. In December 2019, the Company announced a licensing agreement […]

Catabasis: Discontinued Phase III PolarisDMD Trial

Catabasis has announced its Phase 3 PolarisDMD trial discontinuation with edasalonexent, an anti-inflammatory medication under investigation for Duchenne muscular dystrophy. The stop to the PolarisDMD trial was due to a lack of efficacy (no benefit compared to placebo) and not any safety issues. Catabasis is extremely disappointed by this outcome and thanks to the Duchenne […]

Solid Biosciences: Collaboration with Ultragenyx

Solid Biosciences announced a new strategic collaboration with Ultragenyx, a biopharmaceutical company with a successful track record of bringing novel therapies to patients with serious rare and ultra-rare genetic diseases. This new partnership is great news for our community as it will advance meaningful treatment options for Duchenne muscular dystrophy. Solid remains committed to its […]

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