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 (Â鶹Éç) patients with exon duplications. This is the first time a clinical trial has shown production of full-length dystrophin, the protein…
News
The U.S. Food and Drug Administration (FDA) has granted orphan drug status to elamipretide for treating Duchenne muscular dystrophy (Â鶹Éç). 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 (Â鶹Éç). 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 (Â鶹Éç) has launched campaigns through October that involve more than 1,000 retailers nationwide. Proceeds from the Â鶹Éç’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 (Â鶹Éç). 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,…
Astellas Gene Therapies has terminated research and development of its gene therapy programs AT702, AT751, and AT753 for Duchenne muscular dystrophy (Â鶹Éç). The move was based on recent preclinical data, the company announced in a press release. AT702, AT751, and AT753 are exon skipping medicines designed…
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…
Twins Oscar and Sebastian Spink, 11, may not be your typical “Avengers†superheroes, but they are certainly pursuing a gargantuan task that would have impressed even Tony Stark. The boys, diagnosed with facioscapulohumeral muscular dystrophy (FSHD) in 2019 and die-hard fans of the Marvel Cinematic Universe, are attempting to…
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