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Clinical trial strategies for rare neurodevelopmental disorders: challenges and opportunities

Michelle L. Krishnan,Elizabeth Berry-Kravis,Jamie K. Capal,Randall Carpenter,Paul Gringras,Joerg F. Hipp,Meghan T. Miller,Ana Mingorance,Benjamin D. Philpot,Mathew T. Pletcher,Alexander Rotenberg,Jorrit Tjeertes,Paul P. Wang,Tom Willgoss, Marie-Claire de Wit,Shafali S. Jeste

NATURE REVIEWS DRUG DISCOVERY（2021）

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摘要
Novel clinical evaluation strategies are needed to fulfil the potential of targeted therapies for rare neurodevelopmental disorders.
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