Jesse's Journey Foundation

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News & Resources

Muscular Dystrophy Association (MDA): 2021 Conference Update

Jesse’s Journey is pleased to share three treatment updates for Duchenne muscular dystrophy as presented at the recent Muscular Dystrophy Association (MDA) Virtual Clinical & Scientific Conference. Pfizer: PF-06939926 The ongoing Phase 1b study (NCT03362502) is evaluating the safety and tolerability of PF-06939926 in boys who can walk at least 10 meters (about 33 feet) [...]

Solid Biosciences: IGNITE DMD Clinical Trial Update

Solid Biosciences has provided an update on the safety and efficacy data from their IGNITE DMD Phase I/II clinical trial: We are pleased to share an update regarding the safety and efficacy data from our ongoing IGNITE DMD phase I/II clinical trial, as announced in the press release issued this afternoon. Additionally, we are excited to announce […]


COVID-19 and Duchenne

COVID-19 (“coronavirus”) continues to impact our daily lives, and our hearts go out to all those who've been affected. Thank you to all of the health care professionals and everyone on the front lines of this pandemic. Jesse's Journey is here to support the Duchenne community and will continue to provide accurate and timely information [...]

Open Access: Recent studies on Duchenne muscular dystrophy

Recent studies on Duchenne muscular dystrophy (DMD) have greatly deepened our understanding of the primary and secondary pathogenetic mechanisms. Guidelines for the multidisciplinary care for Duchenne that address obtaining a genetic diagnosis and managing the various aspects of the disease have been established. In addition, a number of therapies that aim to restore the missing […]


NS Pharma, Inc.: RACER53 Recruitment

The Hospital for Sick Children (SickKids) in Toronto is currently recruiting boys with Duchenne muscular dystrophy aged 4-7 with mutations amenable to exon 53 skipping for the NS Pharma, Inc. RACER53 clinical trial investigating the new drug, viltolarsen. Click here to learn more and find out if you might qualify for this study: racer53dmdstudy.com. RACER53 Study Overview […]


VISION-DMD: White Paper on Return of Clinical Trial Data

VISION-DMD published a white paper on how to return individual clinical trial results back to patients. It focuses on addressing the ethical and technical challenges, using the vamorolone clinical trial experience. In short: Often, individual patient data is not returned to the participants, even when this is requested. ReveraGen received a grant to develop an [...]

Santhera Pharmaceuticals: VISION-DMD Clinical Trial Update

Santhera Pharmaceuticals has announced the completion of the first 6-month period of  VISION-DMD, a clinical trial investigating vamorolone. If there are positive top-line results in the data, to be released sometime in the second quarter of this year, it could lead to a submission to the Food and Drug Administration (FDA) in the first quarter [...]

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 […]


Disclaimer: Jesse's Journey provides the above information as a potential resource for our visitors. We do not verify the accuracy or truth of third party information and cannot be responsible for the quality of the information or services provided by other organizations and mentioned on our website, nor do we endorse any service, product, treatment or therapy.