‘Life-changing’ Gains Evident in Children Given PTC-AADC Gene Therapy

‘Life-changing’ Gains Evident in Children Given PTC-AADC Gene Therapy

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‘Life-changing’ Gains Evident in Children Given PTC-AADC Gene Therapy

The investigational gene therapy PTC-AADC safely led to profound improvements in children with aromatic L-Amino acid decarboxylase (AADC) deficiency, which was sustained for up to 10 years, its developer, PTC Therapeutics, reported. Treated children developed unheard of cognitive and language skills, and gains in motor function, holding up their head, standing or sitting with support, and communicating. They also had fewer respiratory infections, according to new analysis of five-year data. These findings were presented in a series…

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