Jesse's Journey Foundation

If a once-modest regional hospital and its new biotech allies have their way, the capital of Ohio could one day rival America’s other biomedical hubs. “Our goal is to make Columbus the center of the universe for gene therapy,” says Doug Ingram, CEO of Sarepta Therapeutics. Sarepta (NASDAQ: SRPT) is based in Cambridge, MA, as you […]

Using a genetic engineering tool to restore dystrophin while raising levels of utrophin, a similar protein, leads to better improvements in muscle function than either approach alone, research in a mouse model of Duchenne muscular dystrophy (DMD) reports. The study, “The potential of utrophin and dystrophin combination therapies for Duchenne muscular dystrophy,” appeared in the journal Human Molecular […]

Scientists are testing nearly two dozen treatments that might stop the disease. But enrollment in the trials is very restricted, and few children qualify. Lucas was 5 before his parents, Bill and Marci Barton of Grand Haven, Mich., finally got an explanation for his difficulties standing up or climbing stairs. The diagnosis: muscular dystrophy. Mr. […]

New nine-month data on four boys with Duchenne muscular dystrophy (DMD) enrolled in Study-101 testing Sarepta Therapeutics‘ micro-dystrophin gene therapy continues to show “very encouraging” results, company officials said. These updated data, given in a recent investors’ webinar, show the gene therapy resulted in 81.2% increase in dystrophin protein expression in the muscles, accompanied by marked improvements in boys’ […]

Abstract Mutations in the dystrophin gene cause Duchenne muscular dystrophy (DMD), which is characterized by lethal degeneration of cardiac and skeletal muscles. Mutations that delete exon 44 of the dystrophin gene represent one of the most common causes of DMD and can be corrected in ~12% of patients by editing surrounding exons, which restores the […]

The investigational treatment edasalonexent was well-tolerated, led to no safety issues, and blocked a crucial muscle disease protein in boys with Duchenne muscular dystrophy(DMD), according to part 1 results of a Phase 1/2 trial. The study, “Phase 1 Study of Edasalonexent (CAT-1004), an Oral NF-κB Inhibitor, in Pediatric Patients with Duchenne Muscular Dystrophy,” was published in the Journal of […]

Throughout 2018, Muscular Dystrophy News Today provided daily coverage of new therapeutic strategies, clinical trials, and other topics related to muscular dystrophy (MD). As we look forward to reporting more news to patients, family members, and caregivers dealing with MD in 2019, here are the Top 10 most-read articles of 2018, with a brief description of their relevance to the […]

Amsterdam, February 21, 2019 – Patients with Duchenne muscular dystrophy (DMD) have few treatment options. Medications currently available or in development either target only a subset of DMD patients with a particular genetic mutation or cause significant side effects. The investigational drug edasalonexent, an oral NF-κB inhibitor, has the potential to slow the progression of […]

Preliminary results of a Phase 1/2 study into the gene therapy candidate SGT-001 in Duchenne muscular dystrophy (DMD) patients show low levels of microdystrophin in patients’ muscles after three months of treatment with an initial low dose and continued safety, Solid Bioscience, the therapy’s developer, reported. Continued safety was also evident, the company said in a press release. It added […]