Safety And Improved Efficacy Outcomes In Children With Aadc Deficiency Treated With Agil-Aadc Gene Therapy: Results From Three Clinical Trials

P. W-L Hwu,Y-H Chien,N-C Lee,S-H Tseng, C-H Tai,A. Conway, T. Berner, M. Pykett

ANNALS OF NEUROLOGY(2019)

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Rare Diseases, Movement Disorders, Translational/Experimental Therapeutics
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