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Niels Hendrickx | IAME
Niels Hendrickx Position: Contract researcher / PhD Student Affiliation: Inserm
Team: MOCLID

PhD Thesis

Méthodes statistiques pour obtenir des preuves solides de l'évolution longitudinale et de l'effet du traitement dans des essais cliniques de très petite taille pour l'ataxie multisystémique

School: UPCité

Supervisor: France Mentré & Emmanuelle Comets

Defense date: 2025-11-26

Publications

A Pharmacometrics-Informed Trial Simulation Framework for Optimizing Study Designs for Disease-Modifying Treatments in Rare Neurological Disorders

2025

DOI

PubMed

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CPT: pharmacometrics & systems pharmacology

Ryeznik Y et al.Ryeznik Y, Hilgers RD, Heussen N, Comets E, Mentré F, Hendrickx N, Karlsson MO, Hooker AC, Hamdan A, Chen X, Schüle R, Synofzik M, Sverdlov O

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

2025

DOI

PubMed

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BMC medical research methodology

Hendrickx N et al.Hendrickx N, Mentré F, Hamdan A, Karlsson MO, Hooker AC, Traschütz A, Gagnon C, Schüle R, Synofzik M, Comets E, ARCA Study Group, EVIDENCE-RND consortium

Longitudinal Analysis of Natural History Progression of Rare and Ultra-Rare Cerebellar Ataxias Using Item Response Theory

2024

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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)

2024

DOI

PubMed

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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