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Researchers publish promising results for ultra-rare disease gene therapy method – PMLiVE

Gene therapy method evaluated in seven children with aromatic amino acid decarboxylase deficiency

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Researchers have published some promising results for a new gene therapy method that may offer hope for children born with the ultra-rare genetic disease aromatic amino acid decarboxylase (AADC) deficiency.
The study, conducted by researchers at the Ohio…

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