Gene therapy improves motor and mental function of aromatic l-amino acid decarboxylase deficiency.
Kojima K, Nakajima T, Taga N, Miyauchi A, Kato M, Matsumoto A, Ikeda T, Nakamura K, Kubota T, Mizukami H, Ono S, Onuki Y, Sato T, Osaka H, Muramatsu SI, Yamagata T.
Kojima K, et al. Among authors: nakajima t.
Brain. 2019 Feb 1;142(2):322-333. doi: 10.1093/brain/awy331.
Brain. 2019.
PMID: 30689738
Free PMC article.
Clinical Trial.