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Indication:

Cystic fibrosis, F508del or responsive CFTR mutation, 2 years and older

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As part of conditioning treatment prior to allogeneic hematopoietic stem cell transplantation (alloHSCT) in adult patients with AML or MDS at increased risk for standard conditioning therapies (In combination with fludarabine)

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Age-Related Macular Degeneration

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Diabetic Macular Edema

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Deleterious or suspected deleterious BRCA mutated (germline and/or somatic) metastatic castration resistant prostate cancer (mCRPC) in adult patients who are asymptomatic/mildly symptomatic, and in whom chemotherapy is not clinically indicated. (in combination with prednisone or prednisolone)

Indication:

In combination with endocrine therapy for the adjuvant treatment of adult patients with hormone receptor (HR)-positive, human epidermal growth factor receptor 2 (HER2)-negative, node-positive, early breast cancer at high risk of disease recurrence based on clinicopathological features

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Schizophrenia

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Metastatic Colorectal Cancer (mCRC) patients with left-sided primary tumours that express wild-type RAS (In combination with chemotherapy)

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Hereditary transthyretin-mediated amyloidosis

Indication:

Primary advanced or recurrent mismatch repair deficient (dMMR)/microsatellite instability-high (MSI-H) endometrial cancer in adult patients who are candidates for systemic therapy (in combination with carboplatin and paclitaxel)