Current therapies for Morquio A syndrome and their clinical outcomes.
Kazuki SawamotoYasuyuki SuzukiWilliam G MackenzieMary C TherouxChristian PizarroHiromasa YabeKenji E OriiRobert W MasonTadao OriiShunji Tomatsu
Published in: Expert opinion on orphan drugs (2016)
ERT and HSCT in combination with surgical intervention(s) are a therapeutic option for Morquio A; however, the approach for bone and cartilage lesion remains an unmet challenge.