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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Detalhes bibliográficos
Publicado no:JIMD Rep
Main Authors: Lukina, Elena, Balwani, Manisha, Belmatoug, Nadia, Watman, Nora, Hughes, Derralynn, Gaemers, Sebastiaan J. M., Foster, Meredith C., Lewis, Grace, Peterschmitt, M. Judith
Formato: Artigo
Idioma:Inglês
Publicado em: John Wiley & Sons, Inc. 2020
Assuntos:
Research Reports
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC7802626/
https://ncbi.nlm.nih.gov/pubmed/33473343
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1002/jmd2.12172
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