Author Archives: Steve Bryson PhD

Muscle MRI Scans Can Serve as Biomarker for DMD Progression

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,…

Losmapimod Fails at FSHD Trial’s Primary Goal, But Shows Potential

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…

Cholesterol Metabolism Is Potential Therapeutic Target in DMD

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…

#MDA2021 – DMD Gene Therapy PF-06939926 Continues to Show Promise

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…

#MDA2021 – Gene Therapy SGT-001 Shows Continued Benefit in DMD Boys

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 SGT-001 continues to lead to improvements in physical functioning, disease-related biomarkers, and patient-reported outcomes…