Advocates understand that eteplirsen is not a cure-all for Duchenne muscular dystrophy. But it’s a start for a condition that has no cure. Joseph Gulfo, a biopharmaceutical expert and outspoken critic of the FDA drug approval process, believes that eteplirsen could be used in conjunction with other drugs, either on the market or on the horizon, similar to antiretroviral therapy (ART) for people living with HIV.
“These rare diseases, no one drug is going to cure [them],” Gulfo, a doctor and professor at Fairleigh Dickinson College who specializes in cancer biology, told me. “The best way to get combination therapy is to get these drugs on the market, provided they’re safe and they’re shown to do something. Then you have the ability to really, really make things better.”