First gene therapy for rare form of muscular dystrophy [Video]

The U.S. Food and Drug Administration has given the green light for the first gene therapy that treats a rare form of muscular dystrophy to be used in most people who have the disease and a certain genetic mutation.Related video above: Major drug companies cap asthma medication costsLast year, the drug — Elevidys, from the biotech company Sarepta Therapeutics — was approved to treat only children ages 4 and 5 with Duchenne muscular dystrophy, one of the most severe forms of inherited muscular dystrophies, who have a confirmed mutation in a gene called DMD that is associated with muscle strength.The FDA announced Thursday that it had given traditional approval for Elevidys for ambulatory people 4 and older with a confirmed mutation in the DMD gene and accelerated approval for non-ambulatory people 4 and older with this mutation. There’s not enough data on safety to support its use in children under …