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  • Long-term treatment of patients aged 12 years and older with a confirmed diagnosis of Fabry disease (α-galactosidase A deficiency) who have an amenable mutation.
  • Treatment of severe hepatic veno-occlusive disease (VOD) following haematopoietic stem-cell transplantation (HSCT) therapy
  • The treatment of adults with locally advanced or metastatic cholangiocarcinoma with a fibroblast growth factor receptor 2 (FGFR2) fusion or rearrangement that have progressed after one or more prior line of systemic therapy.
  • 1) The treatment of adults with newly diagnosed, therapy-related acute myeloid leukemia (t-AML) 2) The treatment of adults with newly diagnosed AML with myelodysplasia-related changes (AML-MRC)
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