Jesse's Journey Foundation

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Research Updates

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 […]


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 […]


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 […]


Capricor Recruiting Duchenne Patients for Phase 2 Trial of CAP-1002

Capricor Therapeutics’ Phase 2 clinical trial to evaluate the safety and effectiveness of CAP-1002 in boys and young men with Duchenne muscular dystrophy (DMD) has started recruiting participants. The HOPE-2 trial (NCT03406780) is planned to enroll up to 84 participants ages 10 or older with advanced stages of the disease who have been treated with standard therapies, including glucocorticoids. From […]


Capricor Initiates HOPE-2 Clinical Trial of Duchenne Drug

This morning, Capricor Therapeutics, Inc. announced that the HOPE-2 clinical trial has been initiated at UC Davis Medical Center in Sacramento, CA. The trial will evaluate the efficacy and safety of Capricor’s novel cellular treatment, CAP-1002, in boys and young men with Duchenne muscular dystrophy (DMD), a devastating and life-threatening genetic disorder with few therapeutic […]


Ezutromid Shows Potential in DMD

Phase II study indicates modulating utrophin may reduce muscle inflammation LOS ANGELES — The investigative utrophin modulator ezutromid showed promise in treating Duchenne muscular dystrophy, according to a interim analysis of an open-label, phase II clinical trial. Ezutromid appeared to have significantly deceased inflammation in the calf muscle of Duchenne patients after 24 weeks, reported […]


Edasalonexent Could Reduce Functional Decline in Boys with DMD, MoveDMD Phase 2 Results Suggest

Results of the Phase 2 MoveDMD trial showed that Catabasis Pharmaceuticals’ candidate drug edasalonexent (CAT-1004) can reduce the rate of functional decline in boys ages 4–7 with Duchenne muscular dystrophy (DMD). The recent findings will be subject of a presentation titled, “MoveDMD: Phase 2 Trial of Edasalonexent, an NF-kB Inhibitor, in 4 to 7-Year Old Patients with Duchenne Muscular Dystrophy,” […]