News

Sarepta Therapeutics‘ microdystrophin gene therapy enhanced dystrophin protein expression in the muscles of four boys with Duchenne muscular dystrophy (DMD) enrolled in a Phase 1/2 trial. More importantly, the gene therapy improved the boys’ functional performance. “The encouraging results that we previously saw and reinforced in the fourth…

Half a year has gone by since disgraced pharma executive Martin Shkreli was sentenced to seven years in federal prison for securities and wire fraud while heading San Diego-based Retrophin. As founder and CEO of another company (then known as Turing Pharmaceuticals), in late 2015 Shkreli bought the rights…

One of New England’s top experts in Duchenne muscular dystrophy will give the keynote address at a conference next month dedicated to finding a cure for the neuromuscular disease. Brenda Wong, MD, is the director of the Duchenne program at University of Massachusetts Medical in Worcester, about a 45-minute…

The Greiner family recently held its 10th Miles for Matthew fundraising event for Parent Project Muscular Dystrophy (PPMD) in Greensboro, North Carolina, the nonprofit announced. Determined to continue to make a difference in the life of Matthew Greiner and others who have Duchenne muscular dystrophy (DMD), the…

Long-term use of steroid treatment is linked to increased weight gain in boys with Duchenne muscular dystrophy (DMD) who are unable to walk, a study shows. Recognition of the effect of steroid therapy on patients’ weight and growth progression can have important implications on treatment management and early diagnosis of…

Flagship Biosciences has developed an automated method to analyze muscle fibers (myofibers) and quantify key disease-related biomarkers, such as dystrophin, for Duchenne muscular dystrophy (DMD). The algorithm, known as MuscleMap, was designed to screen muscle tissue samples and detect fluorescent dyes that tag with increased precision the protein…

Catabasis Pharmaceuticals is launching a global Phase 3 trial to evaluate the potential of edasalonexent to treat boys with Duchenne muscular dystrophy (DMD) regardless of mutation type. The soon-to-open PolarisDMD trial is a global and one-year, placebo-controlled study that will assess the safety and efficacy of edasalonexent in boys, ages 4 through…

The U.S. Food and Drug Administration has lifted the clinical hold on a Phase 1/2 trial evaluating Sarepta Therapeutics‘ micro-dystrophin gene therapy candidate for Duchenne muscular dystrophy (DMD), the company announced in a press release. On July 25, the FDA placed the…

On Saturday, coaches with the American Football Coaches Association (AFCA) across the U.S. will display patches to promote the annual Coach To Cure MD initiative during college football games. The fundraising effort, in its 11th consecutive year, has raised almost $2 million to fight Duchenne muscular dystrophy (DMD) and has…

More than 700 medical experts, pharmaceutical executives, patient advocates, and others are expected to converge on Washington, D.C., next month for the 2018 NORD Rare Diseases & Orphan Products Breakthrough Summit. The Oct. 15-16 event, sponsored by the National Organization for Rare Diseases (NORD), takes place at the…