Home infusion program with enzyme replacement therapy for Fabry disease: The experience of a large Italian collaborative group

Home infusion program with enzyme replacement therapy for Fabry disease: The experience of a large Italian collaborative group

Fabry disease (FD) [OMIM 301500] is an X-linked lysosomal storage disorder caused by a deficiency of the lysosomal enzyme alpha-galactosidase A, resulting in progressive multisystem accumulation of globotriaosylceramide (Gb3). Although the introduction of Enzyme Replacement Therapy (ERT) resulted in...

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Bibliographic Details
Main Authors: D. Concolino, L. Amico, M.D. Cappellini, E. Cassinerio, M. Conti, M.A. Donati, F. Falvo, A. Fiumara, M. Maccarone, R. Manna, A. Matucci, M.B. Musumeci, A. Nicoletti, R. Nisticò, F. Papadia, R. Parini, D. Peluso, L. Pensabene, A. Pisani, G. Pistone, M. Rigoldi, I. Romani, M. Tenuta, G. Torti, M. Veroux, E. Zachara
Format: Article
Language:English
Published: Elsevier 2017-09-01
Series:Molecular Genetics and Metabolism Reports
Subjects:
Fabry disease
Enzyme replacement therapy
Home treatment
Adherence
QoL
Online Access:http://www.sciencedirect.com/science/article/pii/S2214426917300332

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http://www.sciencedirect.com/science/article/pii/S2214426917300332

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