A new DRUG TRIALS SNAPSHOT FOR EXONDYS 51 is now available.
EXONDYS 51 is a drug for the treatment of a particular type of Duchenne muscular dystrophy (DMD). It is to be used only in patients who have a specific mutation of the dystrophin gene.
Duchenne muscular dystrophy is a rare disease that affects primarily boys. It is caused by a low level of one muscle protein (dystrophin). The lack of dystrophin causes progressive muscle weakness and premature death. This particular type of DMD disease affects about 13 percent of the population with DMD.
Drug Trials Snapshots provide information about who participated in clinical trials that supported the FDA approval of new drugs. The information provided in these Snapshots also highlights whether there were any differences in the benefits and side effects among sex, race and age groups.