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  Oncology (Rare Cancers)

  Nilotinib – CML

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  Metabolic & Genetic Disorders

  Nitisinone – Hereditary Tyrosinemia Type I

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  Neurology & Neuromuscular Disorders

  Nusinersen – Spinal Muscular Atrophy (SMA)

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  Gastroenterology & Hepatology

  Obeticholic Acid – Primary Biliary Cholangitis

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  Ophthalmology (Rare Eye Disorders)

  Ocriplasmin – Vitreomacular Traction

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  Autoimmune & Immunology

  Omalizumab

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  Neurology & Neuromuscular Disorders

  Onasemnogene Abeparvovec – SMA (Gene Therapy)

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  Endocrine & Rare Hormonal Disorders

  Pasireotide – Cushing’s Disease

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  Oncology Biosimilars

  Pegfilgrastim

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  Nephrology & Hematology

  Pegfilgrastim

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  Endocrine & Rare Hormonal Disorders

  Pegvisomant – Acromegaly

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  Oncology Biosimilars

  Pertuzumab