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…
News
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,…
Astellas Gene Therapies has terminated research and development of its gene therapy programs AT702, AT751, and AT753 for Duchenne muscular dystrophy (DMD). 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…
The European Commission is expected to propose a new governing framework for health data next month, called the European Health Data Space (EHDS), with the aim of connecting national health systems to facilitate secure and efficient transfer of data across systems in different European nations. The move is expected to…
DT-DEC01, Dystrogen Therapeutics’ experimental chimeric cell therapy for Duchenne muscular dystrophy (DMD), appears generally safe and improved muscle strength and motor function in the first three boys enrolled in a Phase 1 clinical trial. Based on these positive results of the therapy’s low dose, with follow-up ranging from one…
The National Organization for Rare Disorders (NORD) has updated its State Report Card to make it more digitally friendly and added telehealth to its categories of rare disease policy issues in a nod to its increased use during the ongoing COVID-19 pandemic. NORD’s report card project began seven…
Recent Posts
- MDA 2026: Early trial of PGN-EDODM1 shows biomarker activity in DM1
- As my FSHD progression worsens, I’m battling feelings of shame
- MDA 2026: Sevasemten halts functional decline in Becker trial
- MDA 2026: Keynote speaker to MDA community: ‘Your voice is powerful’
- Guest Voice: Navigating the windy road of rare disease specialists