High-Dose Ambroxol Therapy in Type 1 Gaucher Disease Focusing on Patients with Poor Response to Enzyme Replacement Therapy or Substrate Reduction Therapy

Ambroxol hydrochloride (ABX), an oral mucolytic drug available over the counter for many years, acts as a pharmacological chaperone for mutant glucocerebrosidase, albeit at higher doses. Proof-of-concept reports have been published over the past decade on all three types of Gaucher disease (GD). Her...

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Detalles Bibliográficos
Autores principales: Istaiti, Majdolen, Frydman, Dafna, Dinur, Tama, Szer, Jeff, Revel-Vilk, Shoshana, Zimran, Ari
Formato: Online Artículo Texto
Lenguaje:English
Publicado: MDPI 2023
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10095311/
https://www.ncbi.nlm.nih.gov/pubmed/37047707
http://dx.doi.org/10.3390/ijms24076732

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10095311/
https://www.ncbi.nlm.nih.gov/pubmed/37047707
http://dx.doi.org/10.3390/ijms24076732

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