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The effectiveness and cost-effectiveness of enzyme and substrate replacement therapies: a longitudinal cohort study of people with lysosomal storage disorders.
Wyatt K, Henley W, Anderson L, Anderson R, Nikolaou V, Stein K, Klinger L, Hughes D, Waldek S, Lachmann R, Mehta A, Vellodi A, Logan S. Wyatt K, et al. Among authors: nikolaou v. Health Technol Assess. 2012;16(39):1-543. doi: 10.3310/hta16390. Health Technol Assess. 2012. PMID: 23089251 Free article.
The NCS-LSD cohort study: a description of the methods and analyses used to assess the long-term effectiveness of enzyme replacement therapy and substrate reduction therapy in patients with lysosomal storage disorders.
Henley WE, Anderson LJ, Wyatt KM, Nikolaou V, Anderson R, Logan S. Henley WE, et al. Among authors: nikolaou v. J Inherit Metab Dis. 2014 Nov;37(6):939-44. doi: 10.1007/s10545-014-9679-6. Epub 2014 Feb 12. J Inherit Metab Dis. 2014. PMID: 24519353
338 results