'Transformational' results for children with fatal rare disorder
A new analysis of five-year data for the gene therapy PTC-AADC shows “profound improvements” in children with aromatic L-Amino acid decarboxylase (AADC) deficiency.
Children treated with PTC-AADC developed motor function and cognitive skills not previously seen, such as holding up their head, sitting or standing with support and communicating.
The benefits described persisted for up to ten years, according to the analysis, which was presented by developer PTC Therapeutics (Nasdaq: PTCT).
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