Orphan & Rare Disease Medicines

Showing 25–36 of 51 results

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  Pulmonology & Cardiology

  Lumacaftor / Ivacaftor – Cystic Fibrosis

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  Pulmonology & Cardiology

  Macitentan – PAH

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  Endocrine & Rare Hormonal Disorders

  Mecasermin – Severe Primary IGF-1 Deficiency

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  Oncology (Rare Cancers)

  Midostaurin – Systemic Mastocytosis

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  Neurology & Neuromuscular Disorders

  Miglustat – Niemann–Pick Disease Type

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  Infectious & Tropical Rare Diseases

  Miltefosine – Leishmaniasis

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  Infectious & Tropical Rare Diseases

  Nifurtimox – Chagas Disease

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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