Article
Auditory N1 event-related potential amplitude is predictive of serum concentration of BPN14770 in fragile x syndrome
2024-06-14
Abstract excerpt
<title>Abstract</title> <p>Fragile X syndrome (FXS) is a rare neurodevelopmental disorder caused by a CGG repeat expansion ≥ 200 repeats in 5’ untranslated region of the FMR1 gene, leading to intellectual disability and cognitive difficulties, including in the domain of communication. A recent phase 2a clinical trial testing BPN14770, a phosphodiesterase 4D inhibitor, showed improved cognition in 30 adult males w...
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Identifiers and source
- Literature Corpus work
- 9ae29cdb-687d-52a2-9085-24f25d162557
- DOI
- 10.21203/rs.3.rs-4474353/v1
