Article

Clonazepam repurposing in ARID1B patients through conventional RCT and N-of-1 Trials - An experimental strategy for orphan disease development

2024-02-12

Abstract excerpt

<h4>Background: </h4> Clinical trials for rare disorders have unique challenges, due to low prevalence, patient phenotype variability, and high expectations. These challenges are highlighted by our study on clonazepam in ARID1B patients, a common cause of intellectual disability. Previous studies on Arid1b haploinsufficient mice showed positive effects of clonazepam on various cognitive aspects. <h4>Methods: </h4...

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Literature Corpus work: 0fc29e3c-d9c5-5f97-8ff1-426900559abd
DOI: 10.21203/rs.3.rs-3937179/v1

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