[HTML][HTML] Clinical outcomes among young patients with Fabry disease who initiated agalsidase beta treatment before 30 years of age: An analysis from the Fabry …
RJ Hopkin, GH Cabrera, JL Jefferies, M Yang… - Molecular Genetics and …, 2023 - Elsevier
Background Clinical manifestations of classic Fabry disease (α-galactosidase A deficiency)
usually occur in childhood, while complications involving major organs typically develop in
adulthood. Outcomes of Fabry-specific treatment among young patients have not been
extensively reported. Our aim was to analyze clinical outcomes among patients aged 5–30
years at initiation of treatment with agalsidase beta using data from the Fabry Registry
(NCT00196742, sponsor: Sanofi). Methods Reported GLA variants were predicted to be …
usually occur in childhood, while complications involving major organs typically develop in
adulthood. Outcomes of Fabry-specific treatment among young patients have not been
extensively reported. Our aim was to analyze clinical outcomes among patients aged 5–30
years at initiation of treatment with agalsidase beta using data from the Fabry Registry
(NCT00196742, sponsor: Sanofi). Methods Reported GLA variants were predicted to be …