Improvement of gastrointestinal symptoms in a significant proportion of male patients with classic Fabry disease treated with agalsidase beta: A Fabry Registry analysis stratified by phenotype
Molecular Genetics and Metabolism Reports(2020)
摘要
•GI symptoms significantly reduced in classic male Fabry patients on agalsidase beta.•Treatment was started long after symptoms began; earlier initiation is recommended.•Fabry-unrelated causes should be excluded if GI symptoms persist on treatment.•GI symptoms were much less prevalent among males with later-onset Fabry disease.
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关键词
Abdominal pain,Agalsidase beta,Diarrhea,Fabry disease,Fabry Registry,Phenotypes
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