Add to ORKGBackground. In Fabry disease, progressive glycolipid ac-cumulation leads to organ damage and early demise, but the incidence of renal, cardiac and cerebrovascular events has not been well characterized. Methods. We conducted a retrospective chart review of 279 affected males and 168 females from 27 sites (USA, Canada, Europe). The pre-defined study endpoints included progression of renal, cardiac and cerebrovascular involve-ment and/or death before the initiation of enzyme replace-ment therapy. Results. The mean rate of estimated glomerular filtration rate (eGFR) decline for patients was −2.93 for males, and −1.02 ml/min/1.73 m2/year for females. Prevalence and severity of proteinuria, baseline eGFR<60ml/min/1.73m
Background/Aims: Fabry disease (FD) is a rare inherited lysosomal storage disease with common and se...
To determine the impact of initiating enzyme replacement therapy (ERT) with agalsidase alfa early in...
Outcomes from 5 years of treatment with agalsidase alfa enzyme replacement therapy (ERT) for Fabry d...
In Fabry disease, progressive glycolipid accumulation leads to organ damage and early demise, but th...
Background. The purpose of this study was to identify determinants of renal disease progression in a...
Background. In Fabry disease, end-stage renal disease (ESRD) and severe neurologic and cardiac compl...
Background. Fabry disease is an X-linked disorder of glycosphingolipid catabolism that is the result...
BACKGROUND: Fabry disease, an X-linked lysosomal storage disorder caused by deficiency of alpha-gala...
BACKGROUND: Fabry disease, an X-linked genetic disorder with deficient alpha-galactosidase A activit...
<div><p>Despite enzyme replacement therapy, disease progression is observed in patients with Fabry d...
Despite enzyme replacement therapy, disease progression is observed in patients with Fabry disease. ...
International audienceCONTEXT: Fabry disease is a rare X-linked genetic disease due to pathogenic va...
Background. In Fabry disease, end-stage renal disease (ESRD) and severe neurologic and cardiac compl...
Fabry disease (FD) is a rare, X-linked disorder caused by mutations in the GLA gene encoding the enz...
Background. Studies on renal function in children with Fabry disease have mainly been done using est...
Background/Aims: Fabry disease (FD) is a rare inherited lysosomal storage disease with common and se...
To determine the impact of initiating enzyme replacement therapy (ERT) with agalsidase alfa early in...
Outcomes from 5 years of treatment with agalsidase alfa enzyme replacement therapy (ERT) for Fabry d...
In Fabry disease, progressive glycolipid accumulation leads to organ damage and early demise, but th...
Background. The purpose of this study was to identify determinants of renal disease progression in a...
Background. In Fabry disease, end-stage renal disease (ESRD) and severe neurologic and cardiac compl...
Background. Fabry disease is an X-linked disorder of glycosphingolipid catabolism that is the result...
BACKGROUND: Fabry disease, an X-linked lysosomal storage disorder caused by deficiency of alpha-gala...
BACKGROUND: Fabry disease, an X-linked genetic disorder with deficient alpha-galactosidase A activit...
<div><p>Despite enzyme replacement therapy, disease progression is observed in patients with Fabry d...
Despite enzyme replacement therapy, disease progression is observed in patients with Fabry disease. ...
International audienceCONTEXT: Fabry disease is a rare X-linked genetic disease due to pathogenic va...
Background. In Fabry disease, end-stage renal disease (ESRD) and severe neurologic and cardiac compl...
Fabry disease (FD) is a rare, X-linked disorder caused by mutations in the GLA gene encoding the enz...
Background. Studies on renal function in children with Fabry disease have mainly been done using est...
Background/Aims: Fabry disease (FD) is a rare inherited lysosomal storage disease with common and se...
To determine the impact of initiating enzyme replacement therapy (ERT) with agalsidase alfa early in...
Outcomes from 5 years of treatment with agalsidase alfa enzyme replacement therapy (ERT) for Fabry d...