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

Istaiti, Majdolen; Frydman, Dafna; Dinur, Tama; Szer, Jeff; Revel-Vilk, Shoshana; Zimran, Ari

Istaiti, Majdolen; Frydman, Dafna; Dinur, Tama; Szer, Jeff; Revel-Vilk, Shoshana; Zimran, Ari.

Afiliação

Istaiti M; Gaucher Unit, Shaare Zedek Medical Center, Jerusalem 9103102, Israel.
Frydman D; Gaucher Unit, Shaare Zedek Medical Center, Jerusalem 9103102, Israel.
Dinur T; Gaucher Unit, Shaare Zedek Medical Center, Jerusalem 9103102, Israel.
Szer J; Peter MacCallum Center, Royal Melbourne Hospital, Department of Medicine, University of Melbourne, Melbourne, VIC 3050, Australia.
Revel-Vilk S; Gaucher Unit, Shaare Zedek Medical Center, Jerusalem 9103102, Israel.
Zimran A; Faculty of Medicine, Hebrew University, Jerusalem 91120, Israel.

Int J Mol Sci ; 24(7)2023 Apr 04.

Article em En | MEDLINE | ID: mdl-37047707

Assuntos

Ambroxol; COVID-19; Doença de Gaucher; Humanos; Doença de Gaucher/tratamento farmacológico; Ambroxol/uso terapêutico; Terapia de Reposição de Enzimas; Vértebras Lombares

Palavras-chave

Gaucher disease (GD); Lyso-Gb1; ambroxol; chaperone therapy; enzyme replacement therapy (ERT); suboptimal response; substrate reduction therapy (SRT)

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Texto completo: 1 Base de dados: MEDLINE Assunto principal: Ambroxol / Doença de Gaucher / COVID-19 Limite: Humans Idioma: En Ano de publicação: 2023 Tipo de documento: Article

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