José Lopes, PhD, managing science editor —

A new hand-held device makes monitoring pulmonary function in adolescents with Duchenne muscular dystrophy (DMD) more convenient and may improve disease management, a study shows. The study, “Home-Based Monitoring of Pulmonary Function in Patients with Duchenne Muscular Dystrophy,” was published in the Journal of Neuromuscular…

Treatment with investigational edasalonexent enabled normal growth, preserved muscle function, and slowed disease progression in boys with Duchenne muscular dystrophy (DMD), according to results of a Phase 1/2 trial and its ongoing extension study. Additionally, a Phase 3 trial called PolarisDMD (NCT03703882) also investigating edasalonexent in boys with DMD…

A Phase 2b trial of the investigational therapy vamorolone is 4-to-6-year-old boys with Duchenne muscular dystrophy (DMD) has begun to enroll patients. Patient recruitment for the international ReveraGen BioPharma-sponsored, double-blind VISION-DMD trial (NCT03439670) is now ongoing. More information on study locations and contacts can be found…

Treatment with investigational cell therapy CAP-1002 improved heart and skeletal muscle function, and reduced cardiac scarring in boys and young men with advanced Duchenne muscular dystrophy (DMD) in a Phase 1/2 trial. Findings also revealed no serious safety issues related to treatment with Capricor Therapeutics’ lead therapy candidate. The…

A Phase 3 trial of edasalonexent, an oral disease-modifying treatment candidate, is enrolling boys with Duchenne muscular dystrophy (DMD) at multiple sites in the U.S. and will soon open sites worldwide, according to Catabasis Pharmaceuticals. Nine sites across the U.S. are now enrolling participants for this one-year,…

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…

CureDuchenne and Avidity Biosciences will host a webinar Friday to brief the Duchenne muscular dystrophy (DMD) community on Avidity’s preclinical development of DMD therapy candidates. The webinar will take place at 4 p.m. EST and will feature Debra Miller, CureDuchenne’s founder and CEO, and Arthur A. Levin,…

Vamorolone, a Duchenne muscular dystrophy (DMD) treatment candidate, showed anti-inflammatory activity and an improved safety profile over the glucocorticoid prednisolone in a Phase 2a clinical trial for boys 4 to 6 years old. Findings from the trial were published in the study, “Phase IIa trial…

Using the gene editing process CRISPR/Cas9 enabled a marked restoration of dystrophin production in various muscles of dogs with Duchenne muscular dystrophy (DMD). The study with that finding, “Gene editing restores dystrophin expression in a canine model of Duchenne muscular dystrophy,” was published in the journal…

Treatment of Duchenne muscular dystrophy (DMD) patients with Raxone (idebenone) delays the need for assisted ventilation by three years, new data from a Phase 3 clinical trial show. Trial results also demonstrate that measuring the air flow speed in and out of the lungs, or peak expiratory flow…