Searchable abstracts of presentations at key conferences on calcified tissues

ba0002p133 | (1) | ICCBH2013

Linear growth over 2 years of velaglucerase alfa therapy in children with type 1 Gaucher disease previously treated with imiglucerase

Zimran Ari , Hughes Derralynn , Elstein Deborah , Smith Laurie , Harmatz Paul , Rhead William , Giraldo Pilar , Mendelsohn Nancy , Park Chan-Hoo , Zahrieh David , Crombez Eric

Objectives: As children with confirmed type 1 Gaucher disease (inherited metabolic disorder) may have linear growth retardation, we evaluated linear growth over 2 years in children enrolled in the interventional study TKT034, in which patients receiving imiglucerase enzyme replacement therapy were switched to velaglucerase alfa.Methods: Trial TKT034 enrolled patients who were ≥2 years of age with type 1 Gaucher disease and stable clinical parameter...