Excerpt from PR Newswire (September 19, 2016):
“The Muscular Dystrophy Association today celebrated news of the U.S. Food and Drug Administration’s decision to grant accelerated approval for eteplirsen, the first disease-modifying drug to treat the most common childhood form of muscular dystrophy.
Accelerated approval of the drug, which will treat a subset of those living with Duchenne muscular dystrophy, is an important step forward in the development of therapies for neuromuscular diseases and marks an historic achievement for the entire DMD community.
‘Today has been a long time in the making,’ said MDA President and CEO Steven M. Derks. ‘This is the outcome MDA dreamed of 25 years ago when it was the first to invest in the breakthrough research that led to development of eteplirsen. Throughout this process we have seen the undeniable strength of our community to rally behind MDA’s commitment to find treatments for our families. This is an important victory, and we are honored to stand shoulder-to-shoulder with everyone who has fought to make this day a reality.'”