Patricia Inácio, PhD, science writer —

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…

Bone health varies greatly among patients with facioscapulohumeral muscular dystrophy (FSHD), according to the results of a small group study, and tests for strength and functionality may identify those with higher risks for decreased bone health. The study “Bone Health in Facioscapulohumeral Muscular Dystrophy: A Cross-Sectional Study” was published…

Administration of Inspra (eplerenone) to young boys with Duchenne muscular dystrophy (DMD) improves their heart function and stabilizes it in older boys, a recent study shows. The study “Eplerenone for early cardiomyopathy in Duchenne muscular dystrophy: results of a two-year open-label extension trial” was published in the Orphanet…