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Evaluating enzyme replacement therapies for Anderson-Fabry disease: commentary on a recent report

Anderson-Fabry disease (AFD) is a rare lysosomal storage disorder. Randomized controlled clinical trials (RCTs) are preferred as the highest category of evidence, but limited availability of robust evidence in rare diseases may necessitate the use of less rigorous evidence. An analysis of cohort stu...

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Bibliografske podrobnosti
izdano v:Genet Mol Biol
Main Authors: Giugliani, Roberto, Westwood, Stephanie, Wellhoefer, Hartmann, Schenk, Jörn, Gurevich, Andrey, Kampmann, Christoph
Format: Artigo
Jezik:Inglês
Izdano: Sociedade Brasileira de Genética 2018
Teme:
Online dostop:https://ncbi.nlm.nih.gov/pmc/articles/PMC6415596/
https://ncbi.nlm.nih.gov/pubmed/30334566
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1590/1678-4685-GMB-2017-0345
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