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News & Resources

Microdystrophin Gene Therapy Shows Promise in Dogs with Duchenne Muscular Dystrophy, Study Shows

Injecting a smaller but functional form of the dystrophin gene, called microdystrophin, into dogs naturally affected by Duchenne muscular dystrophy allowed them to recover muscle strength and stabilized their overall disease symptoms, a new study shows. “This preclinical study demonstrates the safety and efficacy of microdystrophin, and makes it possible to consider developing a clinical trial in patients,” Caroline Le […]

Sarepta Launches Exondys 51 Managed Access Program in Europe, North and South America

Sarepta Therapeutics has partnered with Idis Managed Access, a division of Clinigen Group, to develop a managed access program (MAP) for its lead therapy Exondys 51 (eteplirsen) for the treatment of Duchenne muscular dystrophy (DMD). The MAP was planned to provide early, expanded access to Sarepta’s Exondys 51 to patients who have mutations in the dystrophin gene that can be corrected by elimination of a […]

Translarna Beneficial for Patients in Transitional Stage of Nonsense Mutation Duchenne MD, Study Finds

The latest results of the Phase 3 clinical trial for PTC Therapeutics’ candidate Translarna (ataluren) for the treatment of nonsense mutation Duchenne muscular dystrophy (nmDMD) showed that it can benefit patients who are in a transition stage of the disease and are able to walk between 300 and 400 meters in six minutes. The results were featured in […]

Advocating for Canadians affected by Duchenne

As part of our latest strategic plan, Jesse’s Journey identified a need that we felt we were well-suited to address. Recently, in Europe and the U.S., we have seen new treatments approved for boys with Duchenne and even more that are currently in clinical trials. However, they are very expensive and have had challenges getting […]

Sarepta and Genethon Collaborate on Gene Therapy Research for Duchenne

Today, it was announced that Sarepta Therapeutics, Inc. and Genethon have signed a gene therapy research collaboration to jointly develop treatments for Duchenne muscular dystrophy (DMD). Unlike Sarepta’s current pipeline that is focused on specific dystrophin gene mutations, Genethon’s micro-dystrophin gene therapy approach can target most patients with DMD, and the company has demonstrated proof-of-concept […]

FDA Grants RASRx1902 Orphan Drug Status as Potential Therapy for Duchenne Muscular Dystrophy

The U.S. Food and Drug Administration has granted RASRx1902 orphan drug status to treat Duchenne muscular dystrophy (DMD), says its developer, RASRx of Newport Beach, California. The Orphan Drug status is given to therapies intended to prevent and treat rare life-threatening or chronically debilitating conditions that affect no more than five in 10,000 people, and for which […]

Increasing Levels of Adiponectin Hormone Could Help Reverse Progression of Duchenne MD, Study Suggests

Increasing the levels of a muscle-inflammation-reducing hormone called adiponectin could help reverse the progression of Duchenne muscular dystrophy (DMD), a study suggests. The article in the American Journal of Pathology was titled “Potential Therapeutic Action of Adiponectin in Duchenne Muscular Dystrophy.” Adiponectin can reduce acute and chronic muscle inflammation. Previous studies have shown that increasing its expression […]

MicroRNA-29 overexpression by adeno-associated virus suppresses fibrosis and restores muscle function in combination with micro-dystrophin

Our overall goals include developing meaningful treatment for DMD boys at any stage of disease and one of the challenges to treat DMD is the accumulation of fibrosis or scar tissue as muscle loss progresses.  We have previously demonstrated a way to replace the missing dystrophin gene with a miniature dystrophin using gene therapy.  We […]

PTC Therapeutics Completes Acquisition of Emflaza™ for the Treatment of Duchenne Muscular Dystrophy in the U.S.

SOUTH PLAINFIELD, N.J., April 20, 2017 /PRNewswire/ — PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced it has completed its acquisition of all rights to Emflaza™ (deflazacort) for the treatment of Duchenne muscular dystrophy (DMD) in the U.S. Execution of the asset purchase agreement setting forth the terms of the acquisition was announced on March 16, 2017. […]

Gene-editing alternative corrects Duchenne muscular dystrophy

DALLAS – April 12, 2017 – Using the new gene-editing enzyme CRISPR-Cpf1, researchers at UT Southwestern Medical Center have successfully corrected Duchenne muscular dystrophy in human cells and mice in the lab. The UT Southwestern group had previously used CRISPR-Cas9, the original gene-editing system, to correct the Duchenne defect in a mouse model of the disease and […]

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