One-Shot gene therapy tested to fight devastating muscle disease in boys
NCT ID NCT04240314
Summary
This early-stage trial tested a one-time gene therapy injection for boys with a specific genetic form of Duchenne muscular dystrophy. The goal was to see if the treatment was safe and could help their muscles produce a functional version of a crucial protein called dystrophin. The study involved a very small group of three boys to gather initial safety and biological response data.
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Contacts and locations
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Locations
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Nationwide Children's Hospital
Columbus, Ohio, 43205, United States
Conditions
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