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Research Updates

New Published Study Reports Cardiosphere-Derived Cells Improved Skeletal and Cardiac Muscle Function in Mouse Model of Duchenne Muscular Dystrophy

Cedars-Sinai Researchers Say Findings Lay Groundwork for Testing of CAP-1002 in Patients with Duchenne Muscular Dystrophy LOS ANGELES, Feb. 22, 2018 (GLOBE NEWSWIRE) — Capricor Therapeutics (NASDAQ:CAPR) today announced the online publication in Stem Cell Reports of a new study by researchers at the Smidt Heart Institute at Cedars-Sinai Medical Center, who found that cardiosphere-derived cells (CDCs) improved cardiac […]


Phase 3 Trial of Translarna’s Long-term Efficacy Enrolling Duchenne MD Patients with Nonsense Mutations

PTC Therapeutics is recruiting Duchenne muscular dystrophy (DMD) patients ages 5 and older and with a nonsense mutation for a long-term Phase 3 study to characterize the effects of Translarna (ataluren) on disease progression. The trial (NCT03179631) opened in July 2017 and enrolling patients at eight sites across the U.S., and at others in Australia, Korea and Hong […]


Proposed drug policy changes put sick Canadians at risk, says patient advocate

The Canadian Organization for Rare Disorders represents an estimated one in 12 Canadians living with rare diseases. One of Canada’s leading health policy advisors warns that vulnerable patients will be put at risk if proposed changes to drug pricing go into effect in this country. Dr. Durhane Wong-Rieger, head of the Canadian Organization for Rare […]


Santhera Making Raxone Available to DMD Patients in US Under Expanded Access Program

Santhera Pharmaceuticals recently launched an expanded access program (EAP) to allow certain Duchenne muscular dystrophy (DMD) patients in the U.S. to gain access Raxone (idebenone). Raxone is not approved to treat Duchenne MD by the U.S. Food and Drug Administration (FDA) or the European Medicines Agency (EMA). The BreatheDMD EAP program will allow eligible patients who are age 10 or older and with diminishing […]


New CRISPR Strategy Corrects Wider Range of Mutations Responsible for DMD

A new approach to CRISPR-Cas9 gene-editing technology, called myoediting, successfully restored dystrophin production and contraction force in heart muscle cells of Duchenne muscular dystrophy (DMD) patients. The new strategy, developed by U.S. and German researchers, targets sites located in “hot spots” of mutations along the dystrophin gene, allowing its editing and repair. The study, “Correction of diverse muscular dystrophy […]


Gene Editing Method Corrects Mutations in Duchenne Muscular Dystrophy

Scientists from UT Southwestern Medical Center have developed a CRISPR gene-editing method that has the ability to correct 3,000 mutations that lead to Duchenne muscular dystrophy (DMD). Using heart muscle cells (cardiomyocytes) taken from patients with DMD, the researchers found that a single cut in strategic locations in the DNA could offer an efficient substitute […]


Summit Therapeutics plc : Interim 24-Week Data from PhaseOut DMD

Summit Therapeutics plc(‘Summit’, or ‘the Company’) EZUTROMID SIGNIFICANTLY REDUCED MUSCLE DAMAGE IN DMD PATIENTS IN 24-WEEK INTERIM DATA FROM SUMMIT’S PhaseOut DMD CLINICAL TRIAL Increase in Utrophin Protein Expression Observed Summit Accelerating Preparations for Pivotal Clinical Trial Ezutromid is a Potential Disease-Modifying Treatment for the Entire DMD Patient Population Conference Call Scheduled for 1:00pm GMT […]


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 […]