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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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Détails bibliographiques
Publié dans:Genet Mol Biol
Auteurs principaux: Giugliani, Roberto, Westwood, Stephanie, Wellhoefer, Hartmann, Schenk, Jörn, Gurevich, Andrey, Kampmann, Christoph
Format: Artigo
Langue:Inglês
Publié: Sociedade Brasileira de Genética 2018
Sujets:
Accès en ligne: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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