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Papers matching “ARSACS” 3

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A Pharmacometrics‐Informed Trial Simulation Framework for Optimizing Study Designs for Disease‐Modifying Treatments in Rare Neurological Disorders
2025 CPT Pharmacometrics & Systems Pharmacology article Mathematics Statistical Methods in Clinical Trials Open access

Yevgen V. Ryeznik, R.-D Hilgers, Nicole Maria Heussen, Emmanuelle Comets, France Mentré, Niels Hendrickx, +7 more

3

citations
Capturing clinical progression in multisystemic genetic ataxias: lessons from a prospective study of 884 patients with autosomal recessive or early-onset ataxia
2022 medRxiv preprint Neuroscience Genetic Neurodegenerative Diseases Open access

Andreas Traschütz, Astrid Daniela Adarmes-Gómez, Mathieu Anheim, Jonathan Baets, Bernard Brais, Cynthia Gagnon, +18 more

1

citations
Responsiveness of the Scale for the Assessment and Rating of Ataxia and Natural History in 884 Recessive and Early Onset Ataxia Patients
2023 Annals of Neurology article Neuroscience Genetic Neurodegenerative Diseases Open access

Andreas Traschütz, Astrid Daniela Adarmes-Gómez, Mathieu Anheim, Jonathan Baets, Bernard Brais, Cynthia Gagnon, +18 more

36

citations

3 results