Pharmacologic properties of high-dose ambroxol in four patients with Gaucher disease and myoclonic epilepsy
Sign Up to like & getrecommendations! Published in 2019 at "Journal of Medical Genetics"
DOI: 10.1136/jmedgenet-2019-106132
Abstract: Background Ambroxol (ABX) has been suggested as an augmentative pharmacological agent for neuronopathic Gaucher disease (nGD). This study assessed the long-term safety and efficacy of combined therapy with high-dose ABX and enzyme replacement therapy (ERT)… read more here.
Keywords: gaucher disease; dose abx; high dose; day ... See more keywords