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.
After its cannabidiol gel candidate failed a phase 3 trial in fragile X syndrome, Harmony Biosciences has now paused development of the asset in a different genetic disease. Harmony announced in ...
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