Subject Areas on Research
- Gb(3)/creatinine biomarkers for Fabry disease: issues to consider.
- Interaction of the Shiga-like toxin type 1 B-subunit with its carbohydrate receptor.
- Migalastat improves diarrhea in patients with Fabry disease: clinical-biomarker correlations from the phase 3 FACETS trial.
- Novel quantitative method to evaluate globotriaosylceramide inclusions in renal peritubular capillaries by virtual microscopy in patients with fabry disease.
- Pegunigalsidase alfa, a novel PEGylated enzyme replacement therapy for Fabry disease, provides sustained plasma concentrations and favorable pharmacodynamics: A 1-year Phase 1/2 clinical trial.
- Phenylalanine 30 plays an important role in receptor binding of verotoxin-1.
- Proposed high-risk screening protocol for Fabry disease in patients with renal and vascular disease.
- Safety and pharmacodynamic effects of a pharmacological chaperone on α-galactosidase A activity and globotriaosylceramide clearance in Fabry disease: report from two phase 2 clinical studies.