Impact of Long-Term Enzyme Replacement Therapy on Glucosylsphingosine (Lyso-Gb1) Values in Patients with Type 1 Gaucher Disease: Statistical Models for Comparing Three Enzymatic Formulations

For three decades, enzyme replacement therapy (ERT), and more recently, substrate reduction therapy, have been the standard-of-care for type I Gaucher disease (GD1). Since 2012, three different ERTs have been available. No clinical trial or academic study has ever compared these ERTs beyond one year...

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Detalles Bibliográficos
Autores principales: Dinur, Tama, Grittner, Ulrike, Revel-Vilk, Shoshana, Becker-Cohen, Michal, Istaiti, Majdolen, Cozma, Claudia, Rolfs, Arndt, Zimran, Ari
Formato: Online Artículo Texto
Lenguaje:English
Publicado: MDPI 2021
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8307068/
https://www.ncbi.nlm.nih.gov/pubmed/34299318
http://dx.doi.org/10.3390/ijms22147699

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8307068/
https://www.ncbi.nlm.nih.gov/pubmed/34299318
http://dx.doi.org/10.3390/ijms22147699

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