Jesse's Journey Foundation

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Research Updates

Italfarmaco: Letter to the Duchenne Community

Italfarmaco has shared a letter to the Duchenne community which provides an update about navigating trial visits that cannot be adapted for the home setting: “All of us at Italfarmaco would like to thank the families participating in the Phase 3 EPIDYS trial and in the open-label extension study of givinostat in Duchenne muscular dystrophy […]


PTC Therapeutics: Hosts Call to Review Results of Translarna™ (ataluren)

PTC Therapeutics has shared the results of the dystrophin study (Study 045), which assessed dystrophin levels in patients with nonsense mutation Duchenne muscular dystrophy treated with Translarna™ (ataluren). Click here to see the full press release. The Duchenne community is invited to hear more about the study results on their conference call: When: Thursday, February […]


Santhera Pharmaceuticals: Update on Phase 2B Vamorolone Trial

Santhera Pharmaceuticals has shared an update with the Duchenne community regarding the Phase 2b trial for vamorolone, which the company expects to have data from in the second quarter of 2021. Read the full press release here.  Are you participating in one of the vamorolone clinical trials? Contact Suzanne Gaglianone at Suzanne.gaglianone@reveragen.com to opt-in to […]


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. […]


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 […]