A young man with Duchenne muscular dystrophy (DMD), who was participating in Pfizer’s Phase 1B open-label clinical trial evaluating its gene therapy candidate PF-06939926, has died. The patient has only been identified as a young man who was not able to walk. In a statement, Pfizer expressed…
RGX-202, a one-time experimental gene therapy for the treatment of Duchenne muscular dystrophy (DMD), has been granted orphan drug designation by the U.S. Food and Drug Administration (FDA). The designation is given to investigational therapies to treat rare medical conditions or diseases that affect fewer than 200,000 people…
Translarna (ataluren) treatment delayed the loss of walking abilities by more than five years in boys with Duchenne muscular dystrophy (DMD) who carry nonsense mutations in their DMD gene, according to a study based on real-world data. The investigational therapy also slowed lung function decline by almost two…
MRI scans of muscle can be used as a biomarker to assess disease progression in Duchenne muscular dystrophy (DMD) and to support the evaluation of different therapies in clinical trials, an analysis showed. “The results of this study provide further support for the use of imaging as an objective,…
Oral losmapimod, an investigational treatment for people with facioscapulohumeral muscular dystrophy (FSHD), showed clinically relevant benefits over a placebo on multiple measures of disease progression and patient-reported outcomes after almost a year, according to data from the ReDUX4 Phase 2b clinical trial. But the therapy failed to reach…
An analysis of microRNA molecules isolated from the blood of people with Duchenne muscular dystrophy (DMD) found alterations related to cholesterol metabolism, a study demonstrated. These findings suggest that the cholesterol metabolic pathway may be a potential therapeutic target for this most common type of muscular dystrophy, the…
Top-line data from the VISION-DMD Phase 2b clinical trial evaluating vamorolone in boys with Duchenne muscular dystrophy (DMD), showed efficacy across primary and secondary study goals. The investigational therapy was also well-tolerated with a…
Pfizer has announced a delay in opening U.S. sites for its Phase 3 clinical trial CIFFREO, evaluating the investigational gene therapy PF-06939926 in boys with Duchenne muscular dystrophy (DMD). The delay is caused by questions…
Sarepta Therapeutics has announced positive results from a global study evaluating its investigational treatment SRP-5051 in people with Duchenne muscular dystrophy (DMD)Â who are amenable to exon 51 skipping. Results from part A of the…
Editor’s note: The Muscular Dystrophy News Today team is providing in-depth coverage of the 2021 MDA Virtual Clinical and Scientific Conference, March 15–18. Go here to read the latest stories from the conference. The investigational gene therapy PF-06939926 continues to demonstrate an acceptable safety profile and the potential for…
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