Itraconazole and Lacosamide Combination Therapy Rescues CGG Repeat Expansion Induced Neurotoxicity in Fragile X-Associated Tremor/Ataxia Syndrome.
Carriers of the fragile X messenger ribonucleoprotein 1 (FMR1) gene have an elevated risk of developing Fragile X-associated tremor/ataxia syndrome (FXTAS), a progressive late-onset neurodegenerative disorder. Expansion of CGG trinucleotide repeats within the 5' untranslated region leads to RNA-mediated toxicity and pr...