The efficacy and safety of miglustat in the treatment of GM2 gangliosidosis: a systematic review.
Sign Up to like & getrecommendations! Published in 2023 at "European journal of neurology"
DOI: 10.1111/ene.15871
Abstract: BACKGROUND Since the results of previous studies regarding the safety and efficacy of miglustat in GM2 gangliosidosis (GM2g) were inconsistent, we aimed to assess the miglustat therapy in GM2g patients. METHODS This study followed the… read more here.
Keywords: safety; gm2 gangliosidosis; efficacy; treatment ... See more keywords