Results 81 to 90 of about 167,688 (127)

Safe and Successful Treatment With Agalsidase Beta During Pregnancy in Fabry Disease [PDF]

open access: yes, 2015
WOS: 000363832600010PubMed: 26338166Fabry disease, an X-linked lysosomal storage disorder, is caused by alpha-galactosidase A deficiency and leads to accumulation of glycospinhgolipids in most tissues, with life-theratening consequences in the kidney ...
Bahçebaşı, Zerrin Bicik   +1 more
core  

Clearing of GB3 From Endothelial Vessels of Ocular Conjunctiva in Fabry Patients Treated with Agalsidase-Alfa

open access: yes, 2006
Fabry\u27s disease is an X-linked inborn metabolic error caused by a deficiency of lysosomal ?- galactosidase A. Globotriaosylceramide (Gb3) accumulates in lysosomes of various cells.
R. Ebner; P. Rozenfeld; O. Croxatto; C. Fossati
core  

Long-term effectiveness of agalsidase alfa enzyme replacement in Fabry disease : A Fabry Outcome Survey analysis

open access: yes
Outcomes from 5 years of treatment with agalsidase alfa enzyme replacement therapy (ERT) for Fabry disease in patients enrolled in the Fabry Outcome Survey (FOS) were compared with published findings for untreated patients with Fabry disease.
Beck, Michael   +10 more
core  

Long-term outcomes with agalsidase alfa enzyme replacement therapy : Analysis using deconstructed composite events

open access: yes
Altres ajuts: This work was supported by Shire International GmbH.This is a retrospective analysis of Fabry Outcome Survey data from children/adults (n = 677) receiving agalsidase alfa enzyme replacement therapy for a median of 3 years, examining ...
West, Michael L.   +7 more
core  

Impaired motor unit profile in Fabry disease: A quantitative electrophysiological study. [PDF]

open access: yesClin Neurophysiol Pract
Gunduz A   +4 more
europepmc   +1 more source

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