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Research Updates

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,” […]


#AAN2018 – Early Data Supports Golodirsen in DMD Patients Amenable to Exon 53 Skipping

A potential next-generation therapy, golodirsen (SRP-4053) facilitates and increases dystrophin production in Duchenne muscular dystrophy (DMD) patients, an interim analysis of a Phase 1/2 trial being presented at the 2018 American Academy of Neurology (AAN) Annual Meeting in Los Angeles reports. These findings, in  “Golodirsen Induces Exon Skipping Leading to Sarcolemmal Dystrophin Expression in Duchenne Muscular Dystrophy Patients With Mutations […]


Summit Presents New 24-Week Analyses from PhaseOut DMD at the 2018 American Academy of Neurology Annual Meeting

OXFORD, United Kingdom and CAMBRIDGE, Mass., April 20, 2018 (GLOBE NEWSWIRE) — Summit Therapeutics plc (NASDAQ:SMMT) (AIM:SUMM) announces the presentation of new 24-week interim analyses from PhaseOut DMD, a Phase 2 open-label, multi-centre clinical trial of the utrophin modulator ezutromid in Duchenne muscular dystrophy (‘DMD’), at the 70th American Academy of Neurology Annual Meeting (‘AAN’). These new […]


#AAN2018 – Clinical Trials Re-Analysis Shows Emflaza Potential to Preserve Motor Function in Boys with DMD

Treatment with Emflaza (deflazacort) is better at preserving motor function in patients with Duchenne muscular dystrophy (DMD) than standard of care corticosteroid treatments, according to a re-analysis of data from two clinical trials. The new findings follow a reassessment of pooled data obtained in the placebo group of the Study 007 Phase 2b (NCT00592553) and ACT DMD Phase 3 (NCT01826487) […]


#AAN2018 — Exondys 51 Found to Be Safe in Young Infant with DMD, Preliminary Data Shows

Treatment with Exondys 51 (eteplirsen) was found to be safe and well-tolerated in a very young infant with Duchenne muscular dystrophy (DMD), preliminary data of a follow-up study shows. Initial results of the study will be the subject of a poster presentation at the 2018 Annual Meeting of the American Academy of Neurology (AAN), taking place April 21-27 in Los Angeles. The presentation […]