Orphan & Rare Disease Medicines

Showing 49–51 of 51 results

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  Gastroenterology & Hepatology

  Ursodeoxycholic Acid (specific orphan indication) – Primary Biliary Cholangitis

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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)