Clinical Trial Strategies for Rare Neurodevelopmental Disorders
Novel clinical evaluation strategies are needed to fulfil the potential of targeted therapies for rare neurodevelopmental disorders.
An article by Michelle L. Krishnan1,14, Elizabeth Berry-Kravis2, Jamie K. Capal3, Randall Carpenter4, Paul Gringras5, Joerg F. Hipp1, Meghan T. Miller1,15, Ana Mingorance6, Benjamin D. Philpot7, Mathew T. Pletcher1,16, Alexander Rotenberg8, Jorrit Tjeertes1, Paul P. Wang9,10, Tom Willgoss11, Marie-Claire de Wit12 and Shafali S. Jeste13 for more information.
