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CRISPR-Cas9 corrects Duchenne muscular dystrophy exon 44 deletion mutations in mice and human cells

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 […]


Edasalonexent May Limit Disease Progression in Boys with Duchenne MD, Phase 1/2 Trial Shows

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 […]


Top 10 Muscular Dystrophy Articles of 2018

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 […]


New drug for Duchenne muscular dystrophy clears phase 1 clinical trial testing in boys

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 […]


Gene Therapy SGT-001 Shows Signs of Microdystrophin Production in Muscles of Boys in IGNITE DMD Trial

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 […]


Capricor Resumes Dosing of Enrolled Patients in HOPE-2 Clinical Trial for Duchenne Muscular Dystrophy

LOS ANGELES, Feb. 06, 2019 (GLOBE NEWSWIRE) — Capricor Therapeutics (NASDAQ: CAPR), a clinical-stage biotechnology company, today announced that it has resumed per protocol dosing of patients already enrolled in its HOPE-2 clinical trial of CAP-1002, the company’s novel cell therapy candidate to treat Duchenne muscular dystrophy. Approximately 20 young men and boys in advanced stages of […]


International Preferences for Duchenne Treatments Study

The International Preferences for Duchenne Treatment Study is now open in Canada. Jesse’s Journey has partnered with Johns Hopkins University, Parent Project Muscular Dystrophy, and other Duchenne advocacy organizations from around the world on this important study. This survey is the first international study to measure the preferences and priorities of people with Duchenne and […]


Wave Life Sciences Duchenne Muscular Dystrophy Clinical Trial Selected for FDA Complex Innovative Trial Designs Pilot Program

CAMBRIDGE, Mass., Jan. 03, 2019 (GLOBE NEWSWIRE) — Wave Life Sciences Ltd. (NASDAQ:WVE), a biotechnology company focused on delivering transformational therapies for patients with serious, genetically-defined diseases, today announced that the planned Phase 2/3 efficacy and safety trial for its lead Duchenne muscular dystrophy (DMD) clinical program has been selected for the U.S. Food and […]


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