Compositions and Methods for Treating a Neurodegenerative or Developmental Disorder

Application No.
Broad Case No.

Lindy Barrett, Sara G. Susco, Eugene Yeo, Stefan Aigner, En-Ching Luo


Described and featured are compositions and methods for treating developmental, neurodevelopmental (e.g., Fragile X syndrome (FXS) or Down syndrome (DS)), or neurodegenerative diseases or disorders (e.g., Alzheimer's disease (AD)) by increasing expression of Fragile X Mental Retardation Protein (FMRP) in patients having or having a propensity to develop such diseases or disorders.

Date Published
Date Issued
Published Patent Application
WO 2021/195331
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