Jesse's Journey Foundation

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Research Updates

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 […]

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 […]

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

On May 13, 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 […]

Capricor Therapeutics: CAP1002 Update

Exciting news for Capricor’s CAP1002 therapy for the treatment of cardiomyopathy in advance stage Duchenne muscular dystrophy: On May 13, 2020, Capricor announced positive top-line results from their HOPE-2 study with their lead candidate CAP-1002 treating Duchenne patients with an advanced stage of the disease. CAP1002 is a cardiac-derived cell therapy currently under investigation for […]

Catabasis Connection: May 2020

Catabasis has provided updates on edasalonexent and data from three scientific posters shared during the Muscular Dystrophy Association (MDA) Virtual Poster Session. Click here to see the May 2020 – Catabasis Connection Edasalonexent: In Duchenne, the loss of dystrophin leads to chronic activation of NF-kB, which is a key driver of skeletal and cardiac muscle […]

Italfarmaco: Givinostat Clinical Trial Update

Italfarmaco has provided an update regarding the Givinostat clinical trial for the Duchenne community: The Interim data from EPYDIS Phase 3 trial shows that the trial is not futile and the Independent Data Monitoring Committee recommends trial continuation. Blinded re-estimation of the study sample size allows a reduction of the total number of subjects to […]

Catabasis Connection: Issue 25

Catabasis has shared its March 2020 update: Catabasis provided an update regarding COVID-19 and Duchenne, FAQ’s, and shared the presentation slides from the MDA Virtual Clinical Trials Session. March 2020 – Catabasis Connection Issue 25 Edasalonexent Update:  In Duchenne, the loss of dystrophin leads to chronic activation of NF-kB, which is a key driver of […]