Comprehensive and long-term outcomes of enzyme replacement therapy followed by stem cell transplantation in children with Gaucher disease type 1 and 3.
Usanarat AnurathapanThipwimol Tim-AroonWujuan ZhangWatinee SanpoteSiranee WongrungsriNitcha KhuninSomchai ChutipongtanateVilawan ChirdkiatgumchaiLukana NgiwsaraSuphaneewan JaovisidhaArthaporn KhongkraparnSamart PakakasamaJisnuson SvastiKenneth D R SetchellDuangrurdee WattanasirichaigoonSuradej HongengPublished in: Pediatric blood & cancer (2022)
ERT followed by HSCT could be considered as an alternative treatment for patients with GD3 who have a high risk of fatal neurological progression.