Jesse's Journey Foundation

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Research Updates

Jerry Mendell, in PPMD Webinar, Details Plans for Possible Trial of Duchenne Gene Therapy

In a recent webinar, Dr. Jerry Mendell with Nationwide Children’s Hospital rolled out plans for a Phase 1/2a clinical trial of a new gene therapy for Duchenne’s muscular dystrophy (DMD). Final go-ahead for the trial awaits U.S. Food and Drug Administration (FDA) approval of an Investigational New Drug (IND) application1 made for the therapy and its testing. But Mendell is […]


Phrixus Pharmaceuticals announces the first patient with Duchenne muscular dystrophy (DMD) to meet the 15-month mark on Carmeseal-MD™ (Poloxamer 188 NF)

First patient to be treated with Carmeseal-MD (P-188 NF) outside of the United States through Phrixus’s Expanded Access Program with Ethicor Pharma Ltd. – ANN ARBOR, MI – X September 2017 – Phrixus Pharmaceuticals, Inc. (“Phrixus”), a company focused on therapies for Duchenne muscular dystrophy and heart failure, today announced that the Oirst patient with […]


Researchers Isolate ST2 Protein as Potential Biomarker for Heart Disease in Duchenne Muscular Dystrophy

Researchers have found Duchenne muscular dystrophy (DMD) patients with heart disease have very high levels of ST2, an interleukin 1 receptor-like 1 protein. That makes this molecule a potential biomarker for cardiac disease in such patients. The study by scientists at Children’s National Health System in Washington, D.C., “Interleukin 1 Receptor-Like 1 Protein (ST2) is a Potential Biomarker […]


New Gene Therapy to Fix Dystrophin Deficiency in DMD Shows Promise in Mice, Study Shows

Researchers at the University of Missouri have developed a new method to efficiently deliver the correct form of dystrophin gene to muscles as a way to correct the faulty gene that characterizes Duchenne muscular dystrophy (DMD), a mouse study shows. Their study, “A Five-Repeat Micro-Dystrophin Gene Ameliorated Dystrophic Phenotype in the Severe DBA/2J-mdx Model of Duchenne Muscular Dystrophy,” appeared in […]


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 […]


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 […]