Data/hora: 09/06/2024 05:21:57

Provedor de dados: | 74
País: | Brazil
Título: | Evaluating enzyme replacement therapies for Anderson-Fabry disease: commentary on a recent report
Autores: | Giugliani,Roberto; Westwood,Stephanie; Wellhoefer,Hartmann; Schenk,Jörn; Gurevich,Andrey; Kampmann,Christoph.
Data: | 2018-12-01
Ano: | 2018
Palavras-chave: | Agalsidase alfa; Agalsidase beta; Anderson-Fabry disease; Enzyme replacement therapy.
Resumo: | Abstract 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 studies of enzyme replacement therapies for AFD published in 2017 by El Dib and coworkers made treatment recommendations that contradict previously published findings from RCTs and a systematic Cochrane review. Our commentary outlines concerns regarding selection criteria and statistical methods with their analysis.
Tipo: | Info:eu-repo/semantics/article
Idioma: | Inglês
Identificador: | http://www.scielo.br/scielo.php?script=sci_arttext&pid=S1415-47572018000500790
Editor: | Sociedade Brasileira de Genética
Relação: | 10.1590/1678-4685-gmb-2017-0345
Formato: | text/html
Fonte: | Genetics and Molecular Biology  v.41 n.4 2018
Fonte: | info:eu-repo/semantics/openAccess