Genetic Disorders

Before approving an investigational drug, safety and effectivity needs to be proven. Something different happened with Sarepta’s Muscular dystrophy drug Eteplirsen, as its luck changed completely when the U.S. Food and Drug Administration on 19th September 2016 approved Sarepta Therapeutics' Exondys 51 (eteplirsen) injection to treat patients with Duchenne muscular dystrophy (DMD). Exondys51 is specifically indicated for patients who have a confirmed mutation of the dystrophin gene amenable to exon 51 skipping, which affects about 13 percent of the population with DMD. This is the first drug to get approved for this rare disease. Earlier in March, 2016, FDA had declare...