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Bypassing the metabolic block in an inborn error of vitamin metabolism: a phase 2 clinical trial of 4'-phosphopantetheine in PKAN

P. Hogarth,A. Gregory, S. Jeong, K. Ramsey, A. Freed, P. Rai, K. Wakeman, D. Stone, C. Rogers, H. Hektor,O. Sibon,S. Hayflick

ANNALS OF NEUROLOGY（2023）

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关键词
Experimental Therapeutics, Movement Disorders, Neurometabolic
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