Utilizing real-world evidence to investigate treatments in alternating hemiplegia of childhood: Implications for future trials in rare disease-Reference-Cited by-同舟云学术

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Utilizing real-world evidence to investigate treatments in alternating hemiplegia of childhood: Implications for future trials in rare disease

Published:2024-03 Issue: Volume:49 Page:A1
ISSN:1090-3798
Container-title:European Journal of Paediatric Neurology
language:en
Short-container-title:European Journal of Paediatric Neurology

Author:

Auvin Stéphane^ORCID

Publisher

Elsevier BV

Reference6 articles.

1. Real life retrospective study of cannabidiol therapy in alternating hemiplegia of childhood;Patel;Eur. J. Paediatr. Neurol.,2024

2. Drug development for rare paediatric epilepsies: current state and future directions;Auvin;Drugs,2019

3. Contribution of real-world evidence in European medicines agency's regulatory decision making;Bakker;Clin. Pharmacol. Therapeut.,2023

4. The clinical global impression scale: applying a research tool in clinical practice;Busner;Psychiatry (Edgmont),2007

5. An adapted clinical global Impression of improvement for use in Angelman syndrome: validation analyses utilizing data from the NEPTUNE study;Ventola;Eur. J. Paediatr. Neurol.,2023

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