Work

Clinical trial strategies for rare neurodevelopmental disorders: challenges and opportunities

Year: 2021

Type: article

Source: Nature Reviews Drug Discovery

DOI: 10.1038/d41573-021-00085-9

Authors: 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

Institutions: F. Hoffmann-La Roche AG, Rush University Medical Center, University of North Carolina at Chapel Hill, Rett Syndrome Research Trust, King's College London, Evelina London Children's Healthcare, Audentes Therapeutics (United States), Boston Children's Hospital, Yale University, Simons Foundation, Roche (United Kingdom), Erasmus MC, University of California, Los Angeles

Language: English

Topic: Genetics and Neurodevelopmental Disorders

Subfield: Genetics

Field: Biochemistry, Genetics and Molecular Biology

Domain: Life Sciences

SDG: Good health and well-being

Open Access status: green

Funders: F. Hoffmann-La Roche, National Institute of Neurological Disorders and Stroke, Eunice Kennedy Shriver National Institute of Child Health and Human Development

Awards: Mechanisms of Change with Early Intervention in Tuberous Sclerosis Complex, White Matter Fiber Tract Pathology in a Genetically-Defined Neurodevelopmental Disorder

OpenAlex ID: W3160317720

FWCI: 1.129

Cites: 5

Cited by: 12

Related to: 10