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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 […]


A Note from John Davidson, May 24

20-years-ago today I was in Halifax, Nova Scotia preparing to make the crossing to Prince Edward Island and province number three. Coming into Halifax, the RCMP officer behind me honked his horn to point out the cemetery where some of those who were lost in the 1912 sinking of the Titanic are buried. I had […]


A Note from John Davidson, May 17

20-years-ago today I reached New Glasgow, Nova Scotia on my 38th day on the road. I had arrived in North Sydney, Nova Scotia a week earlier and made my way south along the edge of Lake Bras d’Or, which is actually an inland sea, of partially fresh/salt water in the centre of Cape Breton. With […]


FDA Grants Orphan Drug Designation to Sarconeos for Duchenne Muscular Dystrophy

May 15, 2018 Today, BIOPHYTIS announced that the US Food and Drug administration (FDA) has granted orphan drug designation to its drug candidate, Sarconeos, for Duchenne Muscular Dystrophy (DMD). In a recent comment, Stanislas Veillet, CEO of BIOPHYTIS, stated: “The orphan drug designation that has just been granted by the FDA is a significant milestone for […]


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