Jesse's Journey Foundation

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Research Updates

First Duchenne Muscular Dystrophy Patient Dosed in Systemic Microdystrophin Gene Therapy

HACKENSACK, N.J., Jan. 17, 2018 /PRNewswire-USNewswire/ — Parent Project Muscular Dystrophy (PPMD), a nonprofit organization leading the fight to end Duchenne muscular dystrophy (Duchenne), announced that the first Duchenne patient has been dosed with microdystrophin gene therapy by Dr. Jerry Mendell, Dr. Louise Rodino-Klapac, and their team at Nationwide Children’s Hospital. This trial was funded in part by a $2.2 million grant from […]

FDA Grants Orphan Drug Designation to OPMD Therapy

The US Food and Drug Administration (FDA) has granted Orphan Drug Designation to an oculopharyngeal muscular dystrophy (OPMD) therapy. Benitec Biopharma’s BB-301 therapy, a single vector (gene therapy construct) system, was granted the designation almost exactly a year after receiving the same designation from a European Medicines Agency (EMA) committee. The therapy silences the expression […]

Pharma Giants Form UK-Based ‘Project Hercules’ to Generate Evidence for DMD Treatments

Seven pharmaceutical companies are banding together to sponsor Project Hercules — an initiative that addresses evidence generation for Duchenne muscular dystrophy (DMD) treatments. The project, led by the non-profit organization Duchenne UK, aims to simplify the way evidence is generated for health technology assessments by authorities such as Great Britain’s National Institute for Health and Care Excellence […]

Recent developments in Duchenne muscular dystrophy: facts and numbers

Muscular dystrophies are a clinically and genetically heterogeneous group of skeletal muscle‐wasting diseases. Even for experts in the field of neuromuscular diseases, it is becoming increasingly difficult to distinguish and accurately diagnose all forms of muscular dystrophy on clinical grounds alone, as there is currently a still growing number of different genetic loci for muscular […]

Scientists Develop Skeletal Muscle Using Stem Cells

Researchers have taken a major step towards developing a stem cell replacement therapy for debilitating muscle diseases. A team from UCLA have developed a new strategy to efficiently isolate, mature and transplant skeletal muscle cells created from human pluripotent stem cells, which can produce all cell types of the body. The findings could pave the […]

Investigational Therapy SR8278 Shown to Regenerate Muscle in DMD Mice Study

Scientists at Saint Louis University believe that an investigational therapy called SR8278 holds potential for treating Duchenne muscular dystrophy (DMD) after it was shown to increase muscle function and decrease muscle fibrosis in lab mice. The discovery follows the team’s research of the nuclear receptor REV-ERB, which regulates key processes in the body, including muscle generation, […]

Update from PTC Therapeutics

Dear Duchenne Community, As we enter the holiday season, I wanted to thank the community for all your support throughout the regulatory process for Translarna™ (ataluren) with the FDA. Your voice as passionate advocates has been instrumental in this process. As you know, we filed a Formal Dispute Resolution Request (FDRR) with the FDA a […]

Capricor Set to Launch Phase 2 Trial of Cell Therapy CAP-1002 in Advanced DMD Patients

Capricor Therapeutics has received a green light from the U.S. Food and Drug Administration to launch a Phase 2 study of CAP-1002 cell therapy in children and adults with advanced Duchenne muscular dystrophy. Capricor expects to open the trial to patient enrollment in early 2018 and, if successful, to begin steps needed to receive FDA approval of CAP-1002 […]

Solid Biosciences Readies Clinical Trial of SGT-001, a Potential Duchenne MD Gene Therapy

Solid Biosciences announced the pending start of a first clinical trial to test the company’s leading investigational therapy – called SGT-001, a microdystrophin gene transfer candidate, as a potential treatment for Duchenne muscular dystrophy (DMD). The Phase 1/2 trial, called IGNITE DMD, will soon open for patient enrollment in the United States. “For more than three […]