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News & Resources

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 […]


Defeat Duchenne Family Forum Montréal: New Dates!

Due to COVID-19, the Defeat Duchenne Family Forum presented by PTC Therapeutics and in partnership with La Force DMD has been transformed from a one-day “in-person” program into a series of four virtual events taking place May 20 – 30, 2020. Join us as families and caregivers navigating the Duchenne journey across Canada come together with researchers, clinicians, and […]


Walk to Defeat Duchenne: New Date!

The Walk to Defeat Duchenne presented by Sbenati Dentistry has been rescheduled to Monday, September 7, 2020 (World Duchenne Awareness Day) – this means more time to grow your team and raise vital funds! While making plans during this uncertain time is challenging, we hope you will refocus your efforts online – by encouraging your supporters to […]


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 […]


Santhera Pharmaceuticals: Annual Report

Santhera Pharmaceuticals shared its 2019 Annual Results and Highlights Piepleine Progress for Duchenne muscular dystrophy.  “Santhera is emerging as a leader in addressing rare neuromuscular diseases and 2019 saw us prepare the business for several significant value inflection points this year for our drug candidatesto treat DMD,” said Dario Eklund, CEO of Santhera. “The regulatory […]


Solid Biosciences: IGNITE DMD Clinical Trial Update

Solid Biosciences Inc. has provided an update on the IGNITE DMD phase I/II microdystrophin gene therapy clinical program. As previously reported in late 2019, the IGNITE DMD clinical program was placed on hold. Today, they announced the biomarker data from the third patient dosed in the 2E14 vg/kg dose cohort of IGNITE DMD, including three-month […]


Catabasis Connection: Issue 24

Catabasis has shared its February 2020 update: “February is all about the heart and that’s a big focus for us, too! Because the cardiac function is so vital to those affected by Duchenne, we’re passionate to learn more about the heart and edasalonexent in our Phase 3 PolarisDMD trial. In young boys affected by Duchenne, […]


Rare Disease Day 2020

Did you know approximately 1 in 12 Canadians, two-thirds of them children, are affected by a rare disease? Saturday, February 29, 2020, is Rare Disease Day, and we ask for your help in raising awareness of Duchenne muscular dystrophy. Though Duchenne is the most common fatal genetic disorder diagnosed in childhood, it is considered a […]


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