Background: Enzyme replacement therapy (ERT) with recombinant human a-galactosidase has been available for the treatment of Fabry disease since 2001 in Europe and 2003 in the USA. Treatment outcomes with ERT are dependent on baseline patient characteristics, and published data are derived from heterogeneous study populations.Methods: We conducted a comprehensive systematic literature review of all original articles on ERT in the treatment of Fabry disease published up until January 2017. This article presents the findings in adult male patients.Results: Clinical evidence for the efficacy of ERT in adult male patients was available from 166 publications including 36 clinical trial publications. ERT significantly decreases globotriaosylcerami...
BACKGROUND:Anderson-Fabry disease (AFD) is an X-linked recessive inborn error of glycosphingolipid m...
PURPOSE OF REVIEW: This review explores the clinical and pathological features of Fabry disease. New...
Fabry disease is a rare lysosomal storage disorder caused by mutations in the GLA gene, which, witho...
Background: Enzyme replacement therapy (ERT) with recombinant human a-galactosidase has been availab...
Background: Heterozygous females with Fabry disease have a wide range of clinical phenotypes dependi...
Background: Heterozygous females with Fabry disease have a wide range of clinical phenotypes dependi...
Outcomes from 5 years of treatment with agalsidase alfa enzyme replacement therapy (ERT) for Fabry d...
Background: We analysed 5-year treatment with agalsidase alfa enzyme replacement therapy in patients...
Despite enzyme replacement therapy, disease progression is observed in patients with Fabry disease. ...
Abstract Background Fabry disease (FD) is an X-linked lysosomal storage disorder resulting from the ...
Enzyme replacement therapy (ERT) has been used to treat Fabry disease - a progressive lysosomal stor...
Majid Alfadhel1, Sandra Sirrs21Division of Biochemical Diseases, Department of Paediatrics, BC Child...
To determine the impact of initiating enzyme replacement therapy (ERT) with agalsidase alfa early in...
AbstractBackgroundFabry disease is an X-linked inherited lysosomal storage disease that can be treat...
INTRODUCTION: Fabry disease (FD) is a lysosomal storage disorder resulting in progressive nervous sy...
BACKGROUND:Anderson-Fabry disease (AFD) is an X-linked recessive inborn error of glycosphingolipid m...
PURPOSE OF REVIEW: This review explores the clinical and pathological features of Fabry disease. New...
Fabry disease is a rare lysosomal storage disorder caused by mutations in the GLA gene, which, witho...
Background: Enzyme replacement therapy (ERT) with recombinant human a-galactosidase has been availab...
Background: Heterozygous females with Fabry disease have a wide range of clinical phenotypes dependi...
Background: Heterozygous females with Fabry disease have a wide range of clinical phenotypes dependi...
Outcomes from 5 years of treatment with agalsidase alfa enzyme replacement therapy (ERT) for Fabry d...
Background: We analysed 5-year treatment with agalsidase alfa enzyme replacement therapy in patients...
Despite enzyme replacement therapy, disease progression is observed in patients with Fabry disease. ...
Abstract Background Fabry disease (FD) is an X-linked lysosomal storage disorder resulting from the ...
Enzyme replacement therapy (ERT) has been used to treat Fabry disease - a progressive lysosomal stor...
Majid Alfadhel1, Sandra Sirrs21Division of Biochemical Diseases, Department of Paediatrics, BC Child...
To determine the impact of initiating enzyme replacement therapy (ERT) with agalsidase alfa early in...
AbstractBackgroundFabry disease is an X-linked inherited lysosomal storage disease that can be treat...
INTRODUCTION: Fabry disease (FD) is a lysosomal storage disorder resulting in progressive nervous sy...
BACKGROUND:Anderson-Fabry disease (AFD) is an X-linked recessive inborn error of glycosphingolipid m...
PURPOSE OF REVIEW: This review explores the clinical and pathological features of Fabry disease. New...
Fabry disease is a rare lysosomal storage disorder caused by mutations in the GLA gene, which, witho...