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to improving human health and the quality of life.
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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.
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Treatment of severe hepatic veno-occlusive disease (VOD) following haematopoietic stem-cell transplantation (HSCT) therapy
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Oral Nutrition Supplement for Crohn’s disease from 1 year of age
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Medical nutrition for patients with hypercalcemia
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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.
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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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