Longitudinal Analysis of Natural History Progression of Rare and Ultra-Rare Cerebellar Ataxias Using Item Response Theory
2024
DOI
PubMed
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Clinical Pharmacology and Therapeutics
Hamdan A et al.Hamdan A, Hendrickx N, Hooker AC, Chen X, Comets E, Traschütz A, Schüle R, ARCA Study Group, EVIDENCE‐RND Consortium, Mentré F, Synofzik M, Karlsson MO
Prediction of Individual Disease Progression Including Parameter Uncertainty in Rare Neurodegenerative Diseases: The Example of Autosomal-Recessive Spastic Ataxia Charlevoix Saguenay (ARSACS)
The AAPS journal
Hendrickx N et al.Hendrickx N, Mentré F, Traschütz A, Gagnon C, Schüle R, ARCA Study Group, EVIDENCE-R. N. D. consortium, Synofzik M, Comets E
