Alice Melão,  —

Articles by Alice Melão

Phase 3 Trial of Edasalonexent in Treating DMD Opening Soon, Catabasis Says

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 Stops Phase 2 Clinical Studies of Domagrozumab in DMD Boys

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, Myonexus Collaborating to Advance Gene Therapies for LGMD

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…

FDA Grants RMAT Designation to Capricor Cell Therapy for Duchenne

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.