The designer aminoglycoside NB84 significantly reduces glycosaminoglycan accumulation associated with MPS I-H in the Idua-W392X mouse

► Various aminoglycosides that suppress nonsense mutations with were tested. ► NB54 and NB84 suppressed a nonsense mutation in an MPS I-H mouse. ► NB84 significantly reduced GAG levels in the Idua-W392X mouse.