Topics
MigalastatChemical substance
Fabry diseaseDisease
globotriaosylceramideChemical compound
phenotypeHistoric place
diarrheaDisease
plasmaMilitary unit
peritubular capillaryAnatomical structure
migalastatChemical substance
Purpose: Outcomes in patients with Fabry disease receiving migalastat during the phase 3 FACETS trial (NCT00925301) were evaluated by phenotype. Methods: Data were evaluated in two subgroups of patients with migalastat-amenable GLA variants: “classic phenotype” (n = 14; males with residual peripheral blood mononuclear cell αgalactosidase A <3% normal and multiorgan system involvement) and “other patients” (n = 36; males not meeting classic phenotype criteria and all females). Endpoints included estimated glomerular filtration rate (eGFR), left ventricular mass index (LVMi), Gastrointestinal Symptoms Rating Scale diarrhea subscale (GSRS-D), renal peritubular capillary (PTC) globotriaosylceramide (GL-3) inclusions, and plasma globotriaosylsph...
Abstract Background Fabry disease (FD) is a genetic disorder resulting from deficiency of the lysoso...
Purpose To assess the utility of globotriaosylsphingosine (lyso-Gb(3)) for clinical monitoring of tr...
The effect of migalastat on long-term renal outcomes in enzyme replacement therapy (ERT)-naive and E...
PURPOSE: Outcomes in patients with Fabry disease receiving migalastat during the phase 3 FACETS tria...
BACKGROUND: Fabry disease is a rare, multisystemic disorder caused by GLA gene variants that lead to...
Fabry disease (FD) is a rare X-linked lysosomal storage disorder caused by α-galactosidase A (α-Gal ...
Results from the 18-month randomized treatment period of the phase 3 ATTRACT study demonstrated the ...
The treatment options for Fabry disease (FD) are enzyme replacement therapy (ERT) with agalsidase al...
BACKGROUND: Fabry disease is an X-linked lysosomal storage disorder caused by GLA mutations, resulti...
Fabry disease (FD) is a rare X-linked lysosomal storage disorder caused by α-galactosidase A (α-Gal ...
Fabry's disease (FD) is an X-linked lysosomal storage disorder caused by the deficient activity of t...
Background: Fabry disease is frequently characterized by gastrointestinal symptoms, including diarrh...
Background Fabry disease is frequently characterized by gastrointestinal symptoms, including diarrhe...
PURPOSE: Fabry disease is an X-linked lysosomal storage disorder caused by mutations in the α-galact...
Aims Fabry disease (FD) is an X-linked lysosomal storage disorder caused by a deficiency of the lyso...
Abstract Background Fabry disease (FD) is a genetic disorder resulting from deficiency of the lysoso...
Purpose To assess the utility of globotriaosylsphingosine (lyso-Gb(3)) for clinical monitoring of tr...
The effect of migalastat on long-term renal outcomes in enzyme replacement therapy (ERT)-naive and E...
PURPOSE: Outcomes in patients with Fabry disease receiving migalastat during the phase 3 FACETS tria...
BACKGROUND: Fabry disease is a rare, multisystemic disorder caused by GLA gene variants that lead to...
Fabry disease (FD) is a rare X-linked lysosomal storage disorder caused by α-galactosidase A (α-Gal ...
Results from the 18-month randomized treatment period of the phase 3 ATTRACT study demonstrated the ...
The treatment options for Fabry disease (FD) are enzyme replacement therapy (ERT) with agalsidase al...
BACKGROUND: Fabry disease is an X-linked lysosomal storage disorder caused by GLA mutations, resulti...
Fabry disease (FD) is a rare X-linked lysosomal storage disorder caused by α-galactosidase A (α-Gal ...
Fabry's disease (FD) is an X-linked lysosomal storage disorder caused by the deficient activity of t...
Background: Fabry disease is frequently characterized by gastrointestinal symptoms, including diarrh...
Background Fabry disease is frequently characterized by gastrointestinal symptoms, including diarrhe...
PURPOSE: Fabry disease is an X-linked lysosomal storage disorder caused by mutations in the α-galact...
Aims Fabry disease (FD) is an X-linked lysosomal storage disorder caused by a deficiency of the lyso...
Abstract Background Fabry disease (FD) is a genetic disorder resulting from deficiency of the lysoso...
Purpose To assess the utility of globotriaosylsphingosine (lyso-Gb(3)) for clinical monitoring of tr...
The effect of migalastat on long-term renal outcomes in enzyme replacement therapy (ERT)-naive and E...
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