Literature DB >> 21873040

[Enzyme replacement therapy for lysosomal storage disorders].

V Valayannopoulos1, A Brassier, A Chabli, C Caillaud, M Lemoine, T Odent, J B Arnoux, P de Lonlay.   

Abstract

In the last years, much progress has been achieved in the treatment of lysosomal storage disorders. Until recently only symptomatic treatment was available for the affected patients. Progressively enzyme replacement treatments have been developed for several diseases, namely Gaucher disease, Fabry disease, mucopolysaccharidoses type I, II and VI and Pompe disease. In this review we will summarize the efficacy and safety of these treatments and describe new therapeutic trials for other lysosomal storage disorders or perspectives in the use of currently available treatments.
Copyright © 2011 Elsevier Masson SAS. All rights reserved.

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Year:  2011        PMID: 21873040     DOI: 10.1016/j.arcped.2011.07.002

Source DB:  PubMed          Journal:  Arch Pediatr        ISSN: 0929-693X            Impact factor:   1.180


  3 in total

1.  Substrate and Substrate-Mimetic Chaperone Binding Sites in Human α-Galactosidase A Revealed by Affinity-Mass Spectrometry.

Authors:  Adrian Moise; Stefan Maeser; Stephan Rawer; Frederike Eggers; Mary Murphy; Jeff Bornheim; Michael Przybylski
Journal:  J Am Soc Mass Spectrom       Date:  2016-04-25       Impact factor: 3.109

Review 2.  Gaucher's disease and cancer: a sphingolipid perspective.

Authors:  Brian M Barth; Sriram S Shanmugavelandy; Diana M Tacelosky; Mark Kester; Samy A F Morad; Myles C Cabot
Journal:  Crit Rev Oncog       Date:  2013

Review 3.  The role of serratiopeptidase in the resolution of inflammation.

Authors:  Manju Tiwari
Journal:  Asian J Pharm Sci       Date:  2017-02-01       Impact factor: 6.598

  3 in total

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