News

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 FSHD Society has joined forces with the Critical Path Institute (C-Path) to facilitate the collection of data from people with facioscapulohumeral muscular dystrophy (FSHD) who were given a placebo in previous clinical trials. Provided by sponsors of FSHD trials, the data will be integrated into C-Path’s…

New data from a Phase 1/2 clinical trial of an exon-skipping gene therapy suggest it may help preserve muscle function if delivered early on to Duchenne muscular dystrophy (DMD) patients with exon duplications. This is the first time a clinical trial has shown production of full-length dystrophin, the protein…

The U.S. Food and Drug Administration (FDA) has granted orphan drug status to elamipretide for treating Duchenne muscular dystrophy (DMD). According to Stealth BioTherapeutics, the therapy’s developer, elamipretide has been found to help improve the function of mitochondria — the powerhouses of cells — that…

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…

To help support its popular summer camp and the neuromuscular disease community, the Muscular Dystrophy Association (MDA) has launched campaigns through October that involve more than 1,000 retailers nationwide. Proceeds from the MDA’s summer retail campaigns will benefit research, care, and advocacy for those living with muscular…

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 Russian military plane crash near Tetiana Zamorska’s home in Kyiv, Ukraine, was a sign that it was time for her and her family to leave. The treacherous, 34-hour pilgrimage that ultimately brought the group of eight by car to temporary accommodations in neighboring Poland last month was physically and emotionally difficult,…