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Early initiation of enzyme replacement therapy in classical Fabry disease normalizes biomarkers in clinically asymptomatic pediatric patients.
Kritzer, Amy; Siddharth, Aishwarya; Leestma, Kate; Bodamer, Olaf.
Afiliación
  • Kritzer A; Division of Genetics and Genomics, Department of Pediatrics, Boston Children's Hospital, Boston, MA, United States of America.
  • Siddharth A; Division of Genetics and Genomics, Department of Pediatrics, Boston Children's Hospital, Boston, MA, United States of America.
  • Leestma K; Division of Genetics and Genomics, Department of Pediatrics, Boston Children's Hospital, Boston, MA, United States of America.
  • Bodamer O; Division of Genetics and Genomics, Department of Pediatrics, Boston Children's Hospital, Boston, MA, United States of America.
Mol Genet Metab Rep ; 21: 100530, 2019 Dec.
Article en En | MEDLINE | ID: mdl-31660293

Texto completo: 1 Colección: 01-internacional Banco de datos: MEDLINE Tipo de estudio: Guideline Idioma: En Revista: Mol Genet Metab Rep Año: 2019 Tipo del documento: Article País de afiliación: Estados Unidos

Texto completo: 1 Colección: 01-internacional Banco de datos: MEDLINE Tipo de estudio: Guideline Idioma: En Revista: Mol Genet Metab Rep Año: 2019 Tipo del documento: Article País de afiliación: Estados Unidos