Combined miglustat and enzyme replacement therapy in two patients with type 1 Gaucher disease: two case reports

Combined miglustat and enzyme replacement therapy in two patients with type 1 Gaucher disease: two case reports

Abstract Background Intravenous enzyme replacement therapy is a first-line therapy for Gaucher disease type 1, and substrate reduction therapy represents an oral treatment alternative. Both enzyme replacement therapy and substrate reduction therapy are generally used as monotherapies in Gaucher dise...

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Bibliographic Details
Main Authors: Dominick Amato, Mary Anne Patterson
Format: Article
Language:English
Published: BMC 2018-01-01
Series:Journal of Medical Case Reports
Subjects:
Gaucher disease type 1 (GD1)
Enzyme replacement therapy
Substrate reduction therapy
Combination treatment
Online Access:http://link.springer.com/article/10.1186/s13256-017-1541-7

Internet

http://link.springer.com/article/10.1186/s13256-017-1541-7

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