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

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A new simulation framework leverages pharmacometrics to optimise clinical trial designs for disease-modifying treatments in rare neurological conditions.

The authors present a framework using trial simulations to improve the design of studies targeting rare neurological disorders. By integrating pharmacometric data, the model helps in selecting optimal endpoints and sample sizes to increase the probability of trial success.

Acces this publication (pdf)
Pharmacometrics

Year of publication

2025

Source

CPT: Pharmacometrics & Systems Pharmacology

Link to cite

Acces to Link >

Author

Ryeznik Y, Hilgers RD, Heussen N, Comets E, Mentré F, Hendrickx N, et al.

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