Filter By
Clear all Advanced
I am/have/had
added new label for I am/have/had
more
I am looking for
added new label for I am looking for
more
Advanced Filters
Found 3 clinical trials
A Long Term Follow-Up Study of Fabry Disease Subjects Treated With FLT190

Fabry disease is a rare, X-linked inborn error of glycosphingolipid metabolism caused by an abnormal gene encoding the -galactosidase A (GLA) enzyme.

deficiency
fabry's disease
glycosphingolipids
gene therapy
breakdown
  • 0 views
  • 19 Feb, 2024
Fabry Disease Registry & Pregnancy Sub-registry

The Fabry Registry is an ongoing, international multi-center, strictly observational program that tracks the routine clinical outcomes for patients with Fabry disease, irrespective of treatment status.

fabry's disease
replacement therapy
fabrazyme
alpha-galactosidase
agalsidase beta
  • 0 views
  • 05 Aug, 2024
  • 126 locations
A Global Prospective Observational Study of Women With Fabry Disease and Their Infants During Pregnancy and Breastfeeding

This is a global prospective observational study of women with Fabry disease and their infants during pregnancy and/or breastfeeding. The study will evaluate outcomes of pregnancy and/or breastfeeding in women and infants exposed to migalastat.

fabry's disease
migalastat
  • 0 views
  • 19 Feb, 2024