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Real-Life Experience with Oral Eliglustat in Patients with Gaucher Disease Previously Treated with Enzyme Replacement Therapy

Three types of enzyme replacement therapies (ERTs) and two substrate reduction therapies (SRTs) are approved for symptomatic patients with type 1 Gaucher disease (GD1). Eliglustat is the second SRT approved, yet the first to be approved as first-line therapy for any adult patients with compatible CY...

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Detalles Bibliográficos
Autores principales: Istaiti, Majdolen, Becker-Cohen, Michal, Dinur, Tama, Revel-Vilk, Shoshana, Zimran, Ari
Formato: Online Artículo Texto
Lenguaje:English
Publicado: MDPI 2022
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9659144/
https://www.ncbi.nlm.nih.gov/pubmed/36362492
http://dx.doi.org/10.3390/jcm11216265

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