Jesse's Journey Foundation

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News & Resources

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 [...]

Santhera Pharmaceuticals: Idebenone for Duchenne Update

On June 23, 2020, Santhera Pharmaceuticals announced the UK’s renewal of its early access program for idebenone, a medication used to treat Duchenne patients who are experiencing respiratory function decline, and not taking glucocorticoids. The UK’s Medicines and Health products Regulatory Agency (MHRA) has recognized the positive risk/benefits of idebenone in Duchenne patients. As a […]

Jesse’s Journey: 2019/20 Research Grants Announced

Since 1995, Jesse’s Journey has strongly believed in strong partnerships with academics and clinicians to fund the most promising research for Duchenne muscular dystrophy. This focus and fundraising effort have brought us to granting more than $13.1 million across 45 research projects. We have attracted applications from leading Duchenne researchers from all over the world including the […]

Jesse’s Journey: The loss of an incredible volunteer, Ed Coxworthy

Jesse’s Journey has lost one of its greatest supporters. The man we nicknamed “The King of Newfoundland,” Ed Coxworthy, the motorhome driver during the cross Canada journey in 1998-99 passed away Sunday, June 14, 2020, at his home on Bell Island, Newfoundland. Ed was 80 years old. He may have had a gruff exterior, but [...]

Sarepta Therapeutics: Positive Safety and Efficacy Data from SRP-9001

Sarepta Therapeutics has announced positive results from their gene therapy, SRP-9001, under investigation for Duchenne muscular dystrophy. Preliminary data based upon four boys demonstrated the therapy to be safe and tolerable at one year in their clinical trial. Results were published in JAMA Neurology and further support the potential for SRP-9001 to provide clinically meaningful […]

Nippon Shinyaku: Viltolarsen Update

The European Commission has granted orphan drug designation to viltolarsen, a treatment for people with Duchenne muscular dystrophy (DMD) amenable to exon 53 skipping. See the June 8, 2020, vilolarsen press release here. This compound was approved in Japan in March this year under the brand name Viltepso and is currently under priority review by the FDA in the USA. It […]

Jesse’s Journey: $1M Grant Towards Clinical Trial

Jesse's Journey grants $1M toward the launch of a clinical trial investigating vamolorone for Duchenne muscular dystrophy in Canada. Jesse's Journey, Canada's leader in Duchenne muscular dystrophy funded research, is proud to announce a $1M grant towards a clinical trial evaluating vamorolone for the first time in children 2-4 years of age prior to the [...]

Pfizer: DMD Gene Therapy Continues to Show Promise

On May 15, 2020, Pfizer announced positive results from their phase 1B clinical trial on PF-06939926, an investigational gene therapy under development for Duchenne muscular dystrophy. Preliminary data for 9 boys, still walking, aged 6-12, showed encouraging benefits on a number of parameters with a manageable safety profile. There were a few serious adverse events […]

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