Clinical trial strategies for rare neurodevelopmental disorders: challenges and opportunities

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.

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Bibliographische Detailangaben
Veröffentlicht in:	Nature reviews. Drug discovery 2021-09, Vol.20 (9), p.653-654
Hauptverfasser:	Krishnan, Michelle L., Berry-Kravis, Elizabeth, Capal, Jamie K., Carpenter, Randall, Gringras, Paul, Hipp, Joerg F., Miller, Meghan T., Mingorance, Ana, Philpot, Benjamin D., Pletcher, Mathew T., Rotenberg, Alexander, Tjeertes, Jorrit, Wang, Paul P., Willgoss, Tom, de Wit, Marie-Claire, Jeste, Shafali S.
Format:	Artikel
Sprache:	eng
Schlagworte:	Biomedicine Biotechnology Cancer Research Care and treatment Child development deviations Clinical trials Clinical Trials as Topic - methods Comment Developmental disabilities Humans Medicinal Chemistry Methods Molecular Medicine Nervous system diseases Neurodevelopmental Disorders - genetics Neurodevelopmental Disorders - physiopathology Neurodevelopmental Disorders - therapy Outcome and process assessment (Health Care) Pharmacology/Toxicology Rare diseases Rare Diseases - genetics Rare Diseases - physiopathology Rare Diseases - therapy Research Design
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Beschreibung
Zusammenfassung:	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.
ISSN:	1474-1776 1474-1784
DOI:	10.1038/d41573-021-00085-9