Early initiation of enzyme replacement therapy in classical Fabry disease normalizes biomarkers in clinically asymptomatic pediatric patients

Fabry disease is an X-linked lysosomal storage disorder which often presents with renal, cardiac, gastrointestinal, and nervous system abnormalities. Available enzyme replacement therapies have demonstrated efficacy at significantly reducing elevated biomarkers associated with increased disease acti...

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Detalles Bibliográficos
Autores principales: Kritzer, Amy, Siddharth, Aishwarya, Leestma, Kate, Bodamer, Olaf
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Elsevier 2019
Materias:
Case Report
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6807367/
https://www.ncbi.nlm.nih.gov/pubmed/31660293
http://dx.doi.org/10.1016/j.ymgmr.2019.100530

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