Patricia Inácio, PhD,  science writer—

The U.S. Food and Drug Administration has taken a step that will allow Sarepta Therapeutics to start clinical trials of its Duchenne muscular dystrophy therapy SRP-5051. That move was accepting the company’s Investigational New Drug application for SRP-5051. The therapy, known as a peptide phosphorodiamidate morpholino oligomer, is designed to…

Respiratory activity is significantly impaired in Duchenne muscular dystrophy (DMD), but a new mouse study suggests that enhancing breathing via the brain may improve respiratory capacity. The study “Sensorimotor control of breathing in the mdx mouse model of Duchenne muscular dystrophy” was published in The Journal of Physiology. Respiratory failure…

The newly approved Duchenne muscular dystrophy therapy Exondys 51 (eteplirsen) stays in immune cells several days after leaving the blood, continuing to help regenerate muscle fibers, according to a study in mice. Researchers at the Children’s National Health System in the Washington area said the findings on immune cells known as…

The biotechnology company Capricor Therapeutics recently presented data that support the use of its peripheral intravenous delivery method in the upcoming HOPE-2 clinical trial testing the investigational agent CAP-1002 as a potential therapy for Duchenne muscular dystrophy (DMD). The data were presented during a poster session at the Alliance…

Capricor Therapeutics recently shared an update of the company’s quarterly financial results and operational highlights, including positive results for the ongoing HOPE study with Duchenne muscular dystrophy (DMD) patients. The Phase I/II HOPE study (NCT02485938) is assessing the safety and tolerability of investigational…

The investigational therapy MA-0211, also called MTB-1, a potential treatment for Duchenne Muscular Dystrophy (DMD), was approved for a Phase I clinical trial, developer Mitobridge announced. The trial will evaluate the therapy’s safety and tolerability and determine its pharmacokinetics and pharmacodynamics profile, or its behavior in the body and mechanism…

A comprehensive 12-year genetic and clinical analysis of unrelated patients with congenital muscular dystrophies (CMDs) in the U.K. has confirmed MDC1A as the the most common CMD subtype, and identified 160 new mutations. The study, “Congenital muscular dystrophies in the UK population: Clinical and molecular spectrum of a large…

Overnight use of hand braces preserves hand function and delays the development of shortened muscles or joints in boys who have Duchenne muscular dystrophy (DMD), a study shows. The research, “Evaluation of hand orthoses in Duchenne muscular dystrophy,” was published in the journal Disability and Rehabilitation. Life expectancy in boys…

MoveDMD (NCT02439216) is a three-part, ongoing clinical trial investigating an oral drug — edasalonexent (CAT-1004) — as a potential therapy in boys, ages 4 to 7, with Duchenne muscular dystrophy (DMD). The drug targets NF-kB, a protein activated in DMD and shown to promote the disease by driving inflammation, muscle degeneration,…

RNA sequencing may help to diagnose rare genetic muscle diseases — including muscular dystrophies — in people, according to the results of an international collaboration between several scientists. The study, “Improving genetic diagnosis in Mendelian disease with transcriptome sequencing,” was published in the journal Science Translational Medicine. Several muscle…