ba0002p133 | (1) | ICCBH2013
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...