Enzyme replacement therapy in a child with Gaucher disease.
This case report describes an experience of using recombinant enzyme in a child who was diagnosed as a case of Gaucher disease at the age of 3 years. Regular enzyme replacement therapy has resulted in marked improvement in his hemoglobin level, absolute neutrophil count, platelets and physical growth.
Masood
Yasser
Y
Department of Pediatric Medicine, Children Hospital and Institute of Child Health, Lahore.
Ali
Agha Shabbir
AS
D002648:Child; D005776:Gaucher Disease / Q000097:blood / Q000188:drug therapy*; D005962:Glucosylceramidase / Q000627:therapeutic use*; D006801:Humans; D008297:Male; D011994:Recombinant Proteins / Q000627:therapeutic use
D011994:Recombinant Proteins; D005962:Glucosylceramidase