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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 […]


A Note from John Davidson, May 31

20-years-ago today I was in Sackville, New Brunswick after crossing the Confederation Bridge on my 50th day on the road. This structure, which is 60 metres above sea level at its highest point is the world’s longest bridge over ice-covered water. It’s an engineering marvel, but its operators were not about to cut us any […]


Max’s Big Beer to raise awareness about Duchenne

The Hamilton Spectator Hamilton’s Grain & Grit Beer Co. is teaming up with the local charity event Max’s Big Ride to brew the first ever “Max’s Big Beer.” The limited edition beer will go on sale in June with the hope of raising awareness about Duchenne muscular dystrophy. Fifty cents from every can sold will […]


Walk to Defeat Duchenne 2018

The 2018 Walk to Defeat Duchenne took place on Sunday, May 27 in London’s Springbank Park. More than 150 walkers joined us on a beautiful day to walk the 8.25km route. Some even completed the route four times, equaling the 33km that John Davidson walked every day for 286 days when he crossed Canada in […]


Solid Biosciences Announces New Preclinical Data at the American Society of Gene and Cell Therapy Annual Meeting

– Data Reinforce Potential of SGT-001 as an Important Treatment Candidate for DMD – – Company Continues to Advance Gene Therapy Portfolio – CAMBRIDGE, Mass., May 18, 2018 (GLOBE NEWSWIRE) — Solid Biosciences Inc. (NASDAQ:SLDB) today announced the presentation of new preclinical data from its gene therapy development programs for Duchenne muscular dystrophy (DMD). New […]


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