Jesse's Journey Foundation

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Research Updates

Sarepta Therapeutics: Canadian Clinical Trial Recruitment

Sarepta Therapeutics has provided information on the Essence clinical trial currently recruiting in Canada for boys with deletions amenable to exon 45 and exon 53 skipping. The purpose of this Phase III research study is to evaluate the safety and effectiveness of SRP-4045 and SRP- 4053, Sarepta’s exon 45- and exon 53-skipping investigational drugs. Please […]

Italfarmaco: Update on Givinostat

Italfarmaco delivered a presentation at the International Conference on Duchenne and Becker Muscular Dystrophy where their Chief Medical Officer, Dr. Bettica, presented the long term data following patients who have received givinostat in addition to steroid treatment for seven years: “We are very encouraged to see that the long-term study with Givinostat continues to show […]

Sarepta Therapeutics: FDA Approval of AMONDYS 45™ (casimersen)

Sarepta Therapeutics has announced that the U.S. Food and Drug Administration (FDA) has approved the Amondys 45 (casimersen) for patients amenable to skipping exon 45. This extends their commercial reach to approximately 30% of all Duchenne muscular dystrophy patients. “This is an important day for Sarepta and, far more importantly, for the patients that we […]

Italfarmaco: Letter to the Duchenne Community

Italfarmaco has shared a letter to the Duchenne community which provides an update about navigating trial visits that cannot be adapted for the home setting: “All of us at Italfarmaco would like to thank the families participating in the Phase 3 EPIDYS trial and in the open-label extension study of givinostat in Duchenne muscular dystrophy […]

PTC Therapeutics: Hosts Call to Review Results of Translarna™ (ataluren)

PTC Therapeutics has shared the results of the dystrophin study (Study 045), which assessed dystrophin levels in patients with nonsense mutation Duchenne muscular dystrophy treated with Translarna™ (ataluren). Click here to see the full press release. The Duchenne community is invited to hear more about the study results on their conference call: When: Thursday, February […]

Sarepta Therapeutics: Top-Line Results for Part 1 of Study 102 SRP-9001 Gene Therapy

On January 7, Sarepta Therapeutics announced their topline results from their gene therapy candidate SRP-9001. This study is an ongoing, randomized, double-blinded, placebo-controlled trial evaluating the safety, efficacy and tolerability of a single dose of SRP-9001 in 41 Duchenne patients aged 4-7. The study met its biological primary endpoint of micro-dystrophin expression at 12 weeks. […]

Sarepta Therapeutics: Positive Clinical Results from MOMENTUM – Phase 2 Clinical Trial of SRP-5051

Sarepta has announced encouraging interim results with their SRP-5051 exon skipping molecule for both ambulatory (walking) and non-ambulatory (non-walking) Duchenne patients amenable to exon 51 skipping ages 4-21 years. This new second-generation exon skipping molecule in early clinical trials shows higher tissue exposure than Eteplirsen, with a favourable safety profile. They continue to test higher […]

ReveraGen: Receives $3.3 Million from National Institutes of Health (NIH)

ReveraGen BioPharma, the developer of vamorolone, received a 3.3 million dollar grant from the National Institutes of Health (NIH) to support the commercialization of this safer alternative for corticosteroids treatment. This will advance vamorolone towards FDA submission and commercialization in the USA. This is excellent news for the Duchenne community and is still pending their […]