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My Clinical Pipeline column for Nature Medicine this week examines the state of Sarepta's new gene therapy for Duchenne muscular dystrophy application at the FDA. Whatever its final efficacy turns out to be, the general strategy behind the therapy, an engineered micro-dystrophin is very clever.
It was inspired by the case of a 61 year old patient who had a deletion of almost half of his dystrophin gene and was still alive and walking. Here's that ref, from 1990:
https://www.nature.com/articles/343180a0
And here's my column:
