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Use of Ambroxol as Therapy for Gaucher Disease

IMPORTANCE: Ambroxol was identified as an enhancer of stability and residual activity of several misfolded glucocerebrosidase variants in 2009. OBJECTIVES: To assess hematologic and visceral outcomes, biomarker changes, and safety of ambroxol therapy for patients with Gaucher disease (GD) without di...

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Detalles Bibliográficos
Autores principales:	Zhan, Xia, Zhang, Huiwen, Maegawa, Gustavo H. B., Wang, Yu, Gao, Xiaolan, Wang, Dengbin, Li, Jinning
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	American Medical Association 2023
Materias:	Original Investigation
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10285580/ https://www.ncbi.nlm.nih.gov/pubmed/37342037 http://dx.doi.org/10.1001/jamanetworkopen.2023.19364

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