No justification for very high-dose enzyme therapy for patients with type III Gaucher disease
Author:
Publisher
Wiley
Subject
Genetics(clinical),Genetics
Link
http://www.springerlink.com/index/pdf/10.1007/s10545-007-0783-8
Reference13 articles.
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2. Barton NW, Brady RO, Dambrosia JM, et al (1991) Replacement therapy for inherited enzyme deficiency: macrophage-targeted glucocerebrosidase for Gaucher’s disease. N Engl J Med 324: 1464-470.
3. Beutler E (2006) Lysosomal storage diseases: natural history and ethical and economic aspects. Mol Genet Metab 88: 208-15.
4. Beutler E (2007) Consensus recommendations. Br J Haematol 138(6): 673-75.
5. Cox T, Lachmann R, Hollak C, et al (2000) Novel oral treatment of Gaucher’s disease with N-butyldeoxynojirimycin (OGT 918) to decrease substrate biosynthesis. Lancet 355: 1481-485.
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