Enzyme replacement therapy for treating mucopolysaccharidosis type IVA (Morquio A syndrome): effect and limitations.

Shunji TomatsuKazuki SawamotoTsutomu ShimadaMichael B BoberFrancyne KubaskiEriko YasudaRobert W MasonShaukat KhanCarlos J Alméciga-DíazLuis A BarreraWilliam G MackenzieTadao Orii

Published in: Expert opinion on orphan drugs (2015)

We suggest that ERT is a therapeutic option for MPS IVA, providing a modest effect and the majority of the effects are seen in the soft tissues.

Keyphrases

replacement therapy
smoking cessation
gene expression
case report