Safety and clinical activity of elosulfase alfa in pediatric patients with Morquio A syndrome (mucopolysaccharidosis IVA) less than 5 y.
Simon A JonesMartin BialerRossella PariniKen MartinHui WangKe YangAdam J ShaywitzPaul HarmatzPublished in: Pediatric research (2015)
Early intervention with elosulfase alfa is well-tolerated and produces a decrease in uKS and a trend toward improvement in growth.