Amlexanox provides a potential therapy for nonsense mutations in the lysosomal storage disorder Aspartylglucosaminuria
Keywords: آمکسانکس; AGA; Aspartylglucosaminidase; AGU; Aspartylglucosaminuria; AMX; Amlexanox; Asp-AMC; L-Aspartic acid β-(7-amido-4-methylcoumarin); BBB; Blood brain barrier; LSD; Lysosomal storage disorder; NCL; Neuronal ceroid lipofuscinosis; NMD; Nonsense-mediated decay