Opinion: An FDA pathway can accelerate innovation for Duchenne muscular dystrophy
In human biology, the protein dystrophin is a shining example of Joni Mitchell’s classic line, “you don’t know what you’ve got ’til it’s gone.” Dystrophin stabilizes muscle cells. In its absence, the house of cards comes down.
For my 6-year-old son, Charlie, dystrophin will govern how long he lives. And how much dystrophin he has in his body depends on the ability of drug developers to continue improving it with innovation.
