The Effectiveness of Long-Term Agalsidase Alfa Therapy in the Treatment of Fabry Nephropathy

[摘要] Background and objectives Fabry disease is a rare X-linked disease with multisystemic manifestations. This study investigated the effectiveness of long-term enzyme replacement therapy with agalsidase alfa in Fabry nephropathy treatment.

[学科分类] 泌尿医学

[关键词] Bone marrow necrosis;Sickle cell disease;Hyperhemolysis syndrome