One-Shot gene therapy trial offers hope for boys with devastating muscle disease
NCT ID NCT07429240
Summary
This early-stage trial is testing a new, one-time gene therapy called PBGENE-DMD for boys with Duchenne muscular dystrophy (DMD). The study aims to see if the treatment is safe and if it can correct the underlying genetic cause of the disease by helping muscles produce a needed protein. It will enroll up to 18 boys aged 2 to 7 years old who have specific genetic mutations.
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Conditions
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