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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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  Oncology Biosimilars

  Cetuximab

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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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  Nephrology & Hematology

  Darbepoetin Alfa

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  Oncology (Rare Cancers)

  Dasatinib – CML, Philadelphia chromosome

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  Hematology & Rare Blood Disorders

  Deferasirox – Transfusion-dependent Iron Overload Disorders

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  Oncology Biosimilars

  Denosumab

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  Hematology & Rare Blood Disorders

  Eculizumab – Paroxysmal Nocturnal Hemoglobinuria (PNH), Atypical HUS

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  Neurology & Neuromuscular Disorders

  Edaravone – ALS

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  Infectious & Tropical Rare Diseases

  Eflornithine – African Trypanosomiasis