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Catabasis Pharmaceuticals Announces Plans for Edasalonexent Phase 3 POLARIS DMD Trial in Duchenne Muscular Dystrophy

CAMBRIDGE, MA, July 9, 2018 – Catabasis Pharmaceuticals, Inc. (NASDAQ:CATB), a clinical-stage biopharmaceutical company, today announced plans for the Phase 3 POLARIS DMD trial with edasalonexent in patients with Duchenne muscular dystrophy (DMD). Catabasis plans to initiate the global POLARIS DMD trial in the second half of 2018 with top-line results expected in the second […]


A note from John Davidson, June 27

20-years-ago this week I was making my way through the little villages along Highway 132 on the south shore of the St. Lawrence River. We began the week in Montmagny, about 50 kilometres east of Quebec City. This is where the salt water of the Atlantic Ocean pretty much comes to an end. There is […]


Catabasis Pharmaceuticals Presents New Edasalonexent Clinical Biomarker Data Showing NF-kB Inhibition and Target Engagement in the MoveDMD® Trial

CAMBRIDGE, Mass.–(BUSINESS WIRE)–Catabasis Pharmaceuticals, Inc. (NASDAQ:CATB), a clinical-stage biopharmaceutical company, today reported new NF-kB inhibition biomarker results showing edasalonexent target engagement in the MoveDMD Phase 2 trial and open-label extension in boys affected by Duchenne muscular dystrophy (DMD). NF-kB is a fundamental driver of disease progression in DMD. These results add further support to the significant […]


Max’s Big Ride Crosses the Finish Line

The fourth annual Max’s Big Ride crossed the finish line in Ottawa on July 17, 2018. Andrew Sedmihradsky launched Max’s Big Ride in 2015, and each year since then he and his now 7-year-old son, Max, ride their cargo bike 600km from Hamilton, Ontario to Ottawa, Ontario to raise funds and awareness for Duchenne muscular […]


Early data show Sarepta’s investigational gene therapy led to robust microdystrophin expression in three DMD patients

Shares in Sarepta Therapeutics surged as much as 80 percent Tuesday after the company released preliminary data at an investor event from a Phase I/IIa trial showing that its experimental gene therapy, dubbed AAVrh74.MHCK7, boosted production of a truncated form of a muscle-making protein in three young boys with Duchenne muscular dystrophy (DMD). Specifically, the company reported that all […]


A Note from John Davidson, June 14

20-years-ago today I was in Perth-Andover, New Brunswick alongside the St. John River heading toward Quebec and province number five. Along the Trans-Canada I could hear gunfire through the trees as we passed CFB Gagetown. I just assumed the shots were coming from the practice range and that they weren’t aimed toward the highway! There […]


Clinical Trial Recruiting Patients in Canada

Sarepta Therapeutics is now recruiting patients at two Canadian centres for their ESSENCE clinical trial. ESSENCE Trial The ESSENCE trial is a 96-week, double-blind, placebo-controlled research study to evaluate the effectiveness and safety of investigational products, SRP-4045 and SRP-4053, in patients with Duchenne muscular dystrophy.[1] Canadian Locations The ESSENCE trial is currently recruiting patients at […]


FDA Names Potential FSHD Therapy GBC0905 an Orphan Drug to Speed Its Development

The U.S. Food and Drug Administration (FDA) has granted orphan drug status to GBC0905 as a potential treatment for facioscapulohumeral muscular dystrophy (FSHD),its developer, Genea Biocells, announced. Orphan drug status aims to encourage therapies for rare and serious diseases, through benefits such as seven years of market exclusivity and exemption from FDA application fees. GBC0905 […]


CHMP Adopts Positive Opinion for the Expansion of the Translarna™ (ataluren) Label to Include Patients as Young as 2 Years of Age

– European Commission ratification anticipated in coming months – – Approval of the Translarna annual re-assessment also recommended by CHMP – SOUTH PLAINFIELD, N.J., June 1, 2018 /PRNewswire/ — PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has recommended approval […]


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