Thursday, March 16, 2017
PTC Therapeutics has entered into an asset purchase agreement with Marathon Pharmaceuticals to acquire all rights to Emflaza (deflazacort). Emflaza is the first treatment approved in the U.S. for all Duchenne muscular dystrophy (DMD) patients five years and older, regardless of their genetic mutation. DMD is a rare and fatal genetic disorder that results in progressive muscle weakness from early childhood and leads to premature death due to heart and respiratory failure. Emflaza aligns with PTC’s mission to bring therapies to patients who have rare diseases with limited or no treatment options.