Jesse's Journey Foundation

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News & Resources

Santhera Pharmaceuticals: New Licensing Agreement with ReveraGen Pharma

On September 2, 2020, Santhera announced their new licensing agreement with ReveraGen Pharma, the company up until now, responsible for vamorolone. This agreement offers Santhera the advantage of full control of vamorolone. It allows them to pursue the development, registration, and commercialization of this anti-inflammatory drug candidate more effectively and efficiently. Santhera remains committed to […]


NS Pharma: FDA grants accelerated approval for Viltepso (viltolarsen)

The FDA has granted accelerated approval for Viltepso (viltolarsen) injection for the treatment of Duchenne muscular dystrophy (DMD) in patients with a confirmed mutation of the DMD gene is amenable to exon 53 skipping. Click here to read the full press release. The early clinical data demonstrated an increase in dystrophin production that is reasonably […]


Jesse’s Journey: Brand Awareness Survey

As Jesse’s Journey moves forward with future marketing and communication initiatives, we want to know: What does the Jesse’s Journey "brand" mean to you? We ask that you share your thoughts by completing a short survey - only about 10 minutes -  which will help provide us with insights and direction that will enhance our [...]

Jesse’s Journey + Muscular Dystrophy Canada (MDC): Return to School Webinar

Jesse's Journey and Muscular Dystrophy Canada (MDC) were very pleased to join forces to share information on returning to school for children with neuromuscular disorders. Many parents, caregivers, and guardians face new and difficult choices about how their child will return to school in the fall, such as deciding between in-person, virtual learning and home [...]

Congratulations: Dr. Rudnicki Elected as Fellow of the Royal Society

Jesse’s Journey would like to congratulate Dr. Michael Rudnicki, Chief Scientific Officer of Satellos Bioscience and a Jesse’s Journey research funding recipient, for being elected as a Fellow of the Royal Society, the United Kingdom’s national academy of sciences. The Royal Society is the world’s oldest and most esteemed scientific institution founded in 1660 and […]


Solid Biosciences: Update on SGT-001 Phase 1/2 Clinical Trial

On July 24, 2020, Solid Biosciences announced an update on their phase 1/2 clinical trial on SGT-001, an investigational gene therapy under development for Duchenne. This trial is currently on hold due to some safety concerns, but patients enrolled are continuing to engage in study-related activities. Their clinical biomarker results presented at a recent scientific […]


Catabasis Connection: July 2020

Catabasis has provided an update on their “North Star Ambulatory Assessment in Duchenne Muscular Dystrophy” as presented at the Parent Project Muscular Dystrophy (PPMD) Conference in July 2020. Click here to see the July 2020 – Catabasis Connection Edasalonexent: In Duchenne, the loss of dystrophin leads to chronic activation of NF-kB, which is a key […]


PTC Therapeutics: Translarna (Ataluren) Update

PTC Therapeutics announced the expansion of its European label for translarna (ataluren). Ataluren restores dystrophin production in individuals with Duchenne caused by a nonsense mutation. This treatment is only available in Europe. The label expansion now allows patients who have lost their ability to walk to continue to use ataluren. Read the full press release […]


Sarepta Therapeutics: Casimersen Update

Sarepta Therapeutics has completed a new drug submission to the FDA in the United States for Casimersen, their third exon-skipping medicine for individuals with Duchenne amenable to exon 45. Casimersen is designed for the treatment of exon 45 amenable patients, approximately eight percent of patients with Duchenne. Casimersen is the third exon-skipping medicine using the […]


Jesse’s Journey + Muscular Dystrophy Canada (MDC): Burden of Illness Study

Establishing the burden of illness for a rare disease, like Duchenne muscular dystrophy, is critical for Canada's access to medicines. Jesse's Journey has partially funded a burden of illness study currently underway by Muscular Dystrophy Canada (MDC), and we need your support! Please see the details below and complete the online survey before Monday, July [...]

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