Finally, there’s a drug available to treat Duchenne muscular dystrophy, a rare, fatal genetic muscle-wasting disease that grips roughly 10,000 people, mostly boys, in the U.S., who are unlikely to live into their 30s and typically lose the ability to walk in adolescence.
On Sept. 19, the Food and Drug Administration approved Exondys 51 (eteplirsen) after reviewing a clinical trial and hearing words of support from 50 advocates on behalf of the drug, including families with children living with the disease, experts on the illness at UCLA, and even a few politicians, like Florida senator and former presidential candidate Marco Rubio.
