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

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) in nGD. METHODS: ABX+ERT therapy was admin...

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Detalles Bibliográficos
Autores principales: Kim, Yoon-Myung, Yum, Mi-Sun, Heo, Sun Hee, Kim, Taeho, Jin, Hee Kyung, Bae, Jae-sung, Seo, Go Hun, Oh, Arum, Yoon, Hee Mang, Lim, Hyun Taek, Kim, Hyo-Won, Ko, Tae-Sung, Lim, Hyeong‐Seok, Osborn, Mark J, Tolar, Jakub, Cozma, Claudia, Rolfs, Arndt, Zimran, Ari, Lee, Beom Hee, Yoo, Han-Wook
Formato: Online Artículo Texto
Lenguaje:English
Publicado: BMJ Publishing Group 2020
Materias:
Therapeutics
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7029246/
https://www.ncbi.nlm.nih.gov/pubmed/31649052
http://dx.doi.org/10.1136/jmedgenet-2019-106132

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7029246/
https://www.ncbi.nlm.nih.gov/pubmed/31649052
http://dx.doi.org/10.1136/jmedgenet-2019-106132

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