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Article

Systematic Review of N-of-1 Studies in Rare Genetic Neurodevelopmental Disorders: The Power of 1.

Neurology - 16 Mar 2021

Müller Annelieke R, Brands Marion M M G, van de Ven Peter M, Roes Kit C B, Cornel Martina C, van Karnebeek Clara D M, Wijburg Frits A, Daams Joost G, Boot Erik, van Eeghen Agnies M

Abstract excerpt

OBJECTIVE: To improve the use of N-of-1 studies in rare genetic neurodevelopmental disorders, we systematically reviewed the literature and formulated recommendations for future studies. METHODS: The systematic review protocol was registered in the PROSPERO International Prospective Register of Systematic Reviews (CRD42020154720). EMBASE and MEDLINE were searched for relevant studies. Information was recorded on...

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Identifiers

PMID: 33504638
DOI: 10.1212/WNL.0000000000011597
PMCID: PMC8032375

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