Orphan & Rare Disease Medicines

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  Metabolic & Genetic Disorders

  Agalsidase alfa / beta – Fabry Disease

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  Metabolic & Genetic Disorders

  Alglucosidase alfa – Pompe Disease

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  Pulmonology & Cardiology

  Ambrisentan – PAH

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  Immunology & Inflammatory Rare Diseases

  Anakinra – Rare Autoinflammatory Syndromes

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  Oncology (Rare Cancers)

  Arsenic Trioxide – Acute Promyelocytic Leukemia (APL)

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  Oncology (Rare Cancers)

  Blinatumomab

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  Pulmonology & Cardiology

  Bosentan – Pulmonary Arterial Hypertension (PAH)

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  Oncology (Rare Cancers)

  Brentuximab Vedotin – Hodgkin Lymphoma

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  Immunology & Inflammatory Rare Diseases

  Canakinumab – Cryopyrin-Associated Periodic Syndromes (CAPS)

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  Hematology & Rare Blood Disorders

  Crizanlizumab – Sickle Cell Disease

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  Ophthalmology (Rare Eye Disorders)

  Cysteamine – Corneal Cystinosis

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  Oncology (Rare Cancers)

  Dasatinib – CML, Philadelphia chromosome