Article

Measuring Effectiveness of Treatments in Rare Disorders, Evidence from Clinical Trials in Fragile X Syndrome

2023-10-23

Abstract excerpt

Fragile X Syndrome (FXS) is the leading cause of inherited intellectual disability and autism spectrum disorder (ASD). It results from mutations of the FMR1 gene and the subsequent loss of Fragile X mental disorder 1 protein (FMRP). FXS has been the focus of intense preclinical research leading to several clinical trials. Effect sizes compare the effectiveness of treatments for neurodevelopmental disorders, such a...

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Literature Corpus work: f2b2cc7a-7d4f-5f92-87a9-5df9536bc448
DOI: 10.21203/rs.3.rs-3439158/v1

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