Statistical recommendations for count, binary, and ordinal data in rare disease cross-over trials

Abstract Background Recommendations for statistical methods in rare disease trials are scarce, especially for cross-over designs. As a result various state-of-the-art methodologies were compared as neutrally as possible using an illustrative data set from epidermolysis bullosa research to build reco...

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Hlavní autoři:	Martin Geroldinger, Johan Verbeeck, Andrew C. Hooker, Konstantin E. Thiel, Geert Molenberghs, Joakim Nyberg, Johann Bauer, Martin Laimer, Verena Wally, Arne C. Bathke, Georg Zimmermann
Médium:	Artigo
Jazyk:	Inglês
Vydáno:	BMC 2023-12-01
Edice:	Orphanet Journal of Rare Diseases
Témata:	Cross-over Epidermolysis bullosa simplex Generalized pairwise comparison (GPC) Guidance Model averaging NparLD
On-line přístup:	https://doi.org/10.1186/s13023-023-02990-1
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