Alice Melão,  —

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…

Pfizer is halting the clinical development of domagrozumab (PF-06252616) as treatment for Duchenne muscular dystrophy (DMD) in an ongoing Phase 2 trial. The latest trial data failed to demonstrate domagrozumab’s potential to prevent disease progression or improve patient’s physical capacity, as assessed by mean changes (in seconds) from…

Sarepta Therapeutics has established an exclusive collaboration agreement with Myonexus Therapeutics to develop five new therapies targeting various forms of limb-girdle muscular dystrophy (LGMD). These include three clinical and two preclinical stage programs for the most severe and common forms of LGMD. With this partnership, Sarepta will expand…

Capricor Therapeutics’ Phase 2 clinical trial to evaluate the safety and effectiveness of CAP-1002 in boys and young men with Duchenne muscular dystrophy (DMD) has started recruiting participants. The HOPE-2 trial (NCT03406780) is planned to enroll up to 84 participants ages 10 or older with advanced stages…

The U.S. Food and Drug Administration (FDA) granted orphan drug status to MYO-101, a gene therapy being developed by Myonexus Therapeutics for the treatment of limb girdle muscular dystrophy type 2E (LGMD2E) . The FDA’s orphan drug designation is recognition of the potential of MYO-101 for the treatment of…

Treatment with Emflaza (deflazacort) is better at preserving motor function in patients with Duchenne muscular dystrophy (DMD) than standard of care corticosteroid treatments, according to a re-analysis of data from two clinical trials. The new findings follow a reassessment of pooled data obtained in the placebo group…

Treatment with Exondys 51 (eteplirsen) was found to be safe and well-tolerated in a very young infant with Duchenne muscular dystrophy (DMD), preliminary data of a follow-up study shows. Initial results of the study will be the subject of a poster presentation at the 2018 Annual…

CAP-1002, a cell therapy being developed by Capricor Therapeutics to treat Duchenne muscular dystrophy (DMD), has won a Regenerative Medicine Advanced Therapy (RMAT) designation from the U.S. Food and Drug Administration (FDA). RMAT status is given to regenerative medicine therapies that show potential to treat serious conditions lacking available therapies.