Pharmacologic properties of high-dose ambroxol in four patients with Gaucher disease and myoclonic epilepsy.

Yoon-Myung KimMi-Sun YumSun Hee HeoTaeho KimHee Kyung JinJae-Sung BaeGo Hun SeoArum OhHee Mang YoonHyun Taek LimHyo-Won KimTae-Sung KoHyeong-Seok LimMark J OsbornJakub TolarClaudia CozmaArndt RolfsAri ZimranBeom Hee LeeBeom-Hee Lee

Published in: Journal of medical genetics (2019)

Long-term treatment with high-dose ABX+ERT was safe and might help to arrest the progression of the neurological manifestations in GD.

Keyphrases

high dose
low dose
stem cell transplantation
replacement therapy
cell cycle
cell proliferation
cerebral ischemia