First gene therapy recommended for AADC
NICE's final draft guidance has recommended the first and only gene therapy for children with aromatic L-amino acid decarboxylase (AADC) deficiency.
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NICE's final draft guidance has recommended the first and only gene therapy for children with aromatic L-amino acid decarboxylase (AADC) deficiency.
Upstaza™, approved for UK patients 18 months and over, is the first gene replacement therapy infused directly into the brain for the treatment of AADC deficiency.