Comparing randomized trial designs to estimate treatment effect in rare diseases with longitudinal models: a simulation study showcased by Autosomal Recessive Cerebellar Ataxias using the SARA score

Hendrickx, Niels; Mentre, France; Hamdan, Alzahra; Karlsson, Mats O.; Hooker, Andrew C.; Traschuetz, Andreas; Gagnon, Cynthia; Schuele, Rebecca; Synofzik, Matthis; Comets, Emmanuelle

dc.contributor.author	Synofzik, Matthis
dc.contributor.author	Traschütz, Andreas
dc.contributor.author	Schüle, Rebecca
dc.date.accessioned	2026-03-30T09:54:54Z
dc.date.available	2026-03-30T09:54:54Z
dc.date.issued	2025
dc.identifier.issn	1471-2288
dc.identifier.uri	http://hdl.handle.net/10900/177882
dc.language.iso	en	de_DE
dc.publisher	London : Bmc	de_DE
dc.relation.uri	http://dx.doi.org/10.1186/s12874-025-02626-x	de_DE
dc.subject.ddc	610	de_DE
dc.title	Comparing randomized trial designs to estimate treatment effect in rare diseases with longitudinal models: a simulation study showcased by Autosomal Recessive Cerebellar Ataxias using the SARA score	de_DE
dc.type	Article	de_DE
utue.quellen.id	20251118000000_01318
utue.personen.roh	Hendrickx, Niels
utue.personen.roh	Mentre, France
utue.personen.roh	Hamdan, Alzahra
utue.personen.roh	Karlsson, Mats O.
utue.personen.roh	Hooker, Andrew C.
utue.personen.roh	Traschuetz, Andreas
utue.personen.roh	Gagnon, Cynthia
utue.personen.roh	Schuele, Rebecca
utue.personen.roh	Synofzik, Matthis
utue.personen.roh	Comets, Emmanuelle
dcterms.isPartOf.ZSTitelID	Bmc Medical Research Methodology	de_DE
dcterms.isPartOf.ZS-Issue	Article 179	de_DE
dcterms.isPartOf.ZS-Volume	25 (1)	de_DE
utue.fakultaet	04 Medizinische Fakultät	de_DE