Marisa Wexler, MS,  senior science writer—

The experimental oral therapy EDG-5506 has been generally well-tolerated and lowered markers of muscle damage among men with Becker muscular dystrophy (BMD) in the open-label ARCH clinical trial, early data show. Findings supported the shift to a higher daily treatment dose in study patients. “These interim 2-month ARCH data…

The Muscular Dystrophy Association (MDA) is hosting a one-day games streaming fundraiser, dubbed “MDA Rivals,” this Saturday, June 18. The event, streaming on MDA’s Twitch channel, will feature stars from MDA Let’s Play — a platform that aims to raise funds for MDA and awareness about muscular…

Reduced heart function is associated with a higher rate of mortality among people with Duchenne muscular dystrophy (DMD), a new study reports. Results, however, did not suggest that reduced heart function is an independent predictor of survival for DMD patients. Instead, analyses indicated patients who took diuretics or who…

Treatment with glucocorticoids and heart medicines are key factors in determining the prognosis of Duchenne muscular dystrophy (DMD), according to a study. Results also highlighted genetics as an important factor affecting DMD progression. The paper, “Prognostic indicators of disease progression in Duchenne muscular dystrophy: A literature review…

Despite broad initiatives seeking improvements, the time it takes an individual with Duchenne muscular dystrophy (DMD) to get a correct diagnosis has remained largely unchanged over the past three decades, a study reports. It still takes more than two years, on average, from the time symptoms are first noticed…

Treatment with the breast cancer medicine tamoxifen improved the functionality and survival of heart muscle cells in a cellular model of Duchenne muscular dystrophy (DMD), a study reports. The study, “Tamoxifen treatment ameliorates contractile dysfunction of Duchenne muscular dystrophy stem cell-derived cardiomyocytes on bioengineered substrates,” was published…

The Spectrum Health Helen DeVos Children’s Hospital (HDVCH) in Michigan has been recognized as providing the highest quality of care for patients with Duchenne muscular dystrophy. Located in Grand Rapids, the hospital has become the first in the state to receive a certification from Parent Project Muscular Dystrophy…

An international team of scientists has developed a novel exon-skipping therapy mixture that has the potential to treat more than 40% of people with Duchenne muscular dystrophy (DMD). The therapy was described in a study, “Development of DG9 peptide-conjugated single- and multi-exon skipping therapies for the…

The U.S. Food and Drug Administration (FDA) has given Pfizer the go-ahead to open U.S. sites for the Phase 3 CIFFREO clinical trial, testing the experimental gene therapy PF-06939926 in boys with Duchenne muscular dystrophy (DMD). The FDA had placed a hold on Pfizer’s application to start the trial in…

A new natural history study that seeks to collect information about how Becker muscular dystrophy (BMD) progresses in the absence of treatment is now enrolling. The study is being led by the GRASP (General Resolution and Assessments Solving Phenotypes) consortium and Virginia Commonwealth University, its primary sponsor. Also collaborating…