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  Immunology & Inflammatory Rare Diseases

  Tocilizumab – Castleman’s Disease

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  Oncology Biosimilars

  Trastuzumab

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  Gastroenterology & Hepatology

  Ursodeoxycholic Acid (specific orphan indication) – Primary Biliary Cholangitis

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  Autoimmune & Immunology

  Ustekinumab

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  Oncology (Rare Cancers)

  Vismodegib – Advanced Basal Cell Carcinoma

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  Ophthalmology (Rare Eye Disorders)

  Voretigene Neparvovec – Inherited Retinal Dystrophy (RPE65 Mutation)