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Pregnancy outcome in women with Gaucher disease type 1 who had unplanned pregnancies during eliglustat clinical trials

Gaucher disease type 1 (GD1) is an inherited lysosomal storage disorder caused by deficient enzymatic activity of acid β‐glucosidase, resulting in accumulation of its substrate glucosylceramide, leading to debilitating visceral, hematologic, and skeletal manifestations. Women with GD1 are at increas...

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Detalles Bibliográficos
Autores principales: Lukina, Elena, Balwani, Manisha, Belmatoug, Nadia, Watman, Nora, Hughes, Derralynn, Gaemers, Sebastiaan J. M., Foster, Meredith C., Lewis, Grace, Peterschmitt, M. Judith
Formato: Online Artículo Texto
Lenguaje:English
Publicado: John Wiley & Sons, Inc. 2020
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7802626/
https://www.ncbi.nlm.nih.gov/pubmed/33473343
http://dx.doi.org/10.1002/jmd2.12172