Elizabeth Berry-Kravis – Clinical Trials and Cyclic AMP in Fragile X Syndrome (February 24, 2021)

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Video duration: 00:56:57
Fragile X syndrome (FXS) was thought to be a model neurodevelopmental disorder for the translation of mechanism-targeted treatments from basic neuroscience and animal models to patients. Thus far, however, clinical trials of these approaches have failed. More recent refinements of trial designs and outcome measures have led to a successful phase II trial that targets abnormal cAMP regulation in FXS.

In this lecture, Elizabeth Berry-Kravis will focus on early findings of a cAMP production deficit in cells from FXS, FMR1 gene discovery, understanding of FMRP function and identification of…

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