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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...
Autores principales: | Istaiti, Majdolen, Becker-Cohen, Michal, Dinur, Tama, Revel-Vilk, Shoshana, Zimran, Ari |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
MDPI
2022
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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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