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.
- DOI
- 10.1186/s12874-025-02626-x
- Published
- 2025 Jul 30
- Container
- BMC medical research methodology
- Publisher
- Not recorded
- Open access
- yes
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Cite this work
BibTeX
@article{allodium:10.1186/s12874-025-02626-x,
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.},
author = {Hendrickx N and Mentré F and Hamdan A and Karlsson MO and Hooker AC and Traschütz A and Gagnon C and Schüle R and Synofzik M and Comets E and ARCA Study Group, EVIDENCE-RND consortium},
year = {2025},
journal = {BMC medical research methodology},
doi = {10.1186/s12874-025-02626-x},
url = {https://doi.org/10.1186/s12874-025-02626-x}
}RIS
TY - JOUR TI - 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. AU - Hendrickx N AU - Mentré F AU - Hamdan A AU - Karlsson MO AU - Hooker AC AU - Traschütz A AU - Gagnon C AU - Schüle R AU - Synofzik M AU - Comets E AU - ARCA Study Group, EVIDENCE-RND consortium PY - 2025 JO - BMC medical research methodology DO - 10.1186/s12874-025-02626-x UR - https://doi.org/10.1186/s12874-025-02626-x ER -
APA
N, H., F, M., A, H., MO, K., AC, H., A, T., C, G., R, S., M, S., E, C., & consortium, A. S. G. E. (2025). 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.. BMC medical research methodology. https://doi.org/10.1186/s12874-025-02626-x
Source records
- pubmed · retrieved 2026-11-10T14:14:23.202Z
- europe-pmc · retrieved 2026-11-10T14:14:23.210Z
- doaj · retrieved 2026-11-10T14:14:23.206Z
- hal · retrieved 2026-11-10T14:14:23.265Z