This federal Project Grant award from the National Institute of Child Health and Human Development (NICHD), under the Child Health and Human Development Extramural Research program (CFDA 93.865), provides $189,056 to Neurosant Therapeutics LLC to conduct research on therapeutics that can correct the underlying cause of Smith-Magenis Syndrome (SMS).
The goal of the research is to demonstrate that the expression of the RAI1 gene, which is reduced in individuals with SMS, can be increased using a pharmacological approach. The research aims to correct the transcriptomic defects associated with the reduced RAI1 expression in SMS, which is an autosomal dominant neurodevelopmental disorder. The project will involve laboratory research and studies with people to explore the potential therapeutic solutions over the 2-year award period from July 2024 to June 2026.
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