Blocking the brain protein EPAC2 reverses sensory hypersensitivity, social deficits, and seizures in Fragile X syndrome.
Servier is souping up its neurology pipeline with a clinical-stage candidate sourced from U.K. biotech Kaerus Bioscience for a rare genetic disease. The French pharma is offering as much as $450 ...
Mutations in FMR1, the gene encoding fragile X messenger ribonucleoprotein 1 (FMRP), located at Xq27.3 and discovered in 1991, are the leading single-gene causes of autism and intellectual disability.