STAT April 22, 2024
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.
Charlie has Duchenne muscular dystrophy, a degenerative genetic disease that, until recently, has guaranteed death by early adulthood from cardiac or respiratory failure. Like all people with Duchenne, Charlie’s muscles are not coded to produce dystrophin protein, with catastrophic effects from his first days of life.
Drug discovery efforts...
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