Gaucher Disease.
Hughes DA, Pastores GM.
Hughes DA, et al.
2000 Jul 27 [updated 2023 Dec 7]. In: Adam MP, Feldman J, Mirzaa GM, Pagon RA, Wallace SE, Bean LJH, Gripp KW, Amemiya A, editors. GeneReviews® [Internet]. Seattle (WA): University of Washington, Seattle; 1993–2024.
2000 Jul 27 [updated 2023 Dec 7]. In: Adam MP, Feldman J, Mirzaa GM, Pagon RA, Wallace SE, Bean LJH, Gripp KW, Amemiya A, editors. GeneReviews® [Internet]. Seattle (WA): University of Washington, Seattle; 1993–2024.
PMID: 20301446
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MANAGEMENT: Targeted therapy: Options include enzyme replacement therapy (ERT) or substrate reduction therapy (SRT; e.g., miglustat, eliglustat). Hematopoietic stem cell transplantation may be an option in individuals with severe GD, primarily those with chronic neurologic …
MANAGEMENT: Targeted therapy: Options include enzyme replacement therapy (ERT) or substrate reduction therapy (SRT; e.g., miglustat, elig …