Clinical trial strategies for rare neurodevelopmental disorders: challenges and opportunities.

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

Clinical trial strategies for rare neurodevelopmental disorders: challenges and opportunities.

2021

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

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

Keywords:

Intensive care medicine
Clinical trial
clinical evaluation
Medicine

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