Author Archives: Steve Bryson PhD

FDA Places Hold on DYNE-251 Clinical Trial Launch in DMD

The U.S. Food and Drug Administration (FDA) has placed a hold on Dyne Therapeutics’ application to launch a clinical trial evaluating DYNE-251, an experimental exon-skipping therapy for Duchenne muscular dystrophy (DMD). Dyne now hopes to have that Phase 1/2 trial underway by June, the company said in a …

DMD Gene Therapy Candidate RGX-202 Earns Orphan Drug Status

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 Preserved Walking 5-plus Years in DMD Boys

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…

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…