Doctors test First-Ever prenatal gene therapy for devastating baby disease
NCT ID NCT07479953
Summary
This is a first-in-human safety study testing a gene therapy given to babies before they are born. It is for fetuses diagnosed with GM1 gangliosidosis, a rare and severe genetic disorder that damages the brain and body. Doctors will inject a modified virus carrying a healthy gene into the fetus's bloodstream to see if it is safe for both the mother and baby.
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Conditions
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