The FDA recently informed Muscular Dystrophy News that it has granted orphan drug designation to the experimental drug ISX9-CPC, a product of the IPS Heart company located in Houston, Texas.…
Emflaza (deflazacort), a corticosteroid, was originally approved by the FDA in 2017 to treat Duchenne muscular dystrophy (DMD) in patients five years or older. This approval was expanded in 2019…
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