The efficacy and safety of miglustat in the treatment of GM2 gangliosidosis: a systematic review.

Although miglustat should not be considered a definite treatment for GM2g, it seems that patients particularly those with infantile or late-infantile GM2g could benefit from miglustat therapy to some extent. We also suggested some hints for future studies to present their findings in a standard method to make it more possible to pool the available data in such rare diseases for a more comprehensive conclusion.