Long-term effectiveness of enzyme replacement therapy in Fabry disease: results from the NCS-LSD cohort study-Reference-Cited by-同舟云学术

Long-term effectiveness of enzyme replacement therapy in Fabry disease: results from the NCS-LSD cohort study

Published:2014-05-15 Issue:6 Volume:37 Page:969-978
ISSN:0141-8955
Container-title:Journal of Inherited Metabolic Disease
language:en
Short-container-title:J Inherit Metab Dis

Author:

Anderson L. J.,Wyatt K. M.,Henley W.,Nikolaou V.,Waldek S.,Hughes D. A.,Pastores G. M.,Logan S.

Publisher

Wiley

Subject

Genetics (clinical),Genetics

Link

http://link.springer.com/content/pdf/10.1007/s10545-014-9717-4

Reference40 articles.

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2. Banikazemi M, Bultas J, Waldek S et al (2007) Agalsidase-beta therapy for advanced Fabry disease: a randomized trial. Ann Intern Med 146(2):77–86

3. Beck M, Ricci R, Widmer U et al (2004) Fabry disease: overall effects of agalsidase alfa treatment. Eur J Clin Invest 34(12):838–844

4. Connock M, Juarez-Garcia A, Frew E, et al (2006) A systematic review of the clinical effectiveness and cost-effectiveness of enzyme replacement therapies for Fabry's disease and mucopolysaccharidosis type 1. Health Technol Assess 10(20): iii-iv, ix-113

5. Desnick RJ, Wasserstein MP (2001) Fabry disease: clinical features and recent advances in enzyme replacement therapy. Adv Nephrol Necker Hosp 31:317–339

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