[Enzyme replacement therapy for lysosomal storage disorders]

V Valayannopoulos; A Brassier; A Chabli; C Caillaud; M Lemoine; T Odent; J B Arnoux; P de Lonlay

doi:10.1016/j.arcped.2011.07.002

[Enzyme replacement therapy for lysosomal storage disorders]

Arch Pediatr. 2011 Oct;18(10):1119-23. doi: 10.1016/j.arcped.2011.07.002. Epub 2011 Aug 27.

[Article in French]

Authors

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

Affiliation

¹ Centre de référence des maladies héréditaires du métabolisme de l'enfant et de l'adulte (MaMEA), hôpital Necker-Enfants Malades, 149, rue de Sèvres, 75743 Paris cedex 15, France. [email protected]

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

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.

Publication types

Review

MeSH terms

Clinical Trials as Topic
Enzyme Replacement Therapy* / methods
Enzyme Therapy
Enzymes / genetics
Fabry Disease / drug therapy
Gaucher Disease / drug therapy
Glycogen Storage Disease Type II / drug therapy
Humans
Lysosomal Storage Diseases / diagnosis
Lysosomal Storage Diseases / drug therapy*
Lysosomal Storage Diseases / enzymology
Mucopolysaccharidoses / drug therapy
Treatment Outcome

Substances

Enzymes