Pharmaceutical therapies to recode nonsense mutations in inherited diseases
Keywords: 2-DOS, 2-deoxystreptamine; 6MWD, 6-minute walk test; aa-tRNA, aminoacyl-tRNA; ADME, absorption, distribution, metabolism and excretion; AHC, anterior horn cell; ALT, alanine aminotransferase; A-T, ataxia-telangiectasia; ATM, A-T mutated; BBB, blood–brain