Comprehensive and long-term outcomes of enzyme replacement therapy followed by stem cell transplantation in children with Gaucher disease type 1 and 3

Comprehensive and long-term outcomes of enzyme replacement therapy followed by stem cell transplantation in children with Gaucher disease type 1 and 3

Anurathapan U, Tim-Aroon T, Zhang W, Sanpote W, Wongrungsri S, Khunin N, Chutipongtanate S, Chirdkiatgumchai V, Ngiwsara L, Jaovisidha S, Khongkraparn A, Pakakasama S, Svasti J, Setchell KDR, Wattanasirichaigoon D, Hongeng S

Pediatr Blood Cancer. 2023;70(3):e30149.