Orphan & Rare Disease Medicines

Showing 37–48 of 51 results

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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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  Endocrine & Rare Hormonal Disorders

  Pegvisomant – Acromegaly

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  Hematology & Rare Blood Disorders

  Ravulizumab – PNH

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  Neurology & Neuromuscular Disorders

  Riluzole – Amyotrophic Lateral Sclerosis (ALS)

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  Hematology & Rare Blood Disorders

  Romiplostim – ITP

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  Immunology & Inflammatory Rare Diseases

  Ruxolitinib – Graft-Versus-Host Disease

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  Oncology (Rare Cancers)

  Sonidegib – Advanced Basal Cell Carcinoma

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  Oncology (Rare Cancers)

  Sonidegib – Advanced Basal Cell Carcinoma

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  Pulmonology & Cardiology

  Tafamidis – Transthyretin Amyloid Cardiomyopathy

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  Neurology & Neuromuscular Disorders

  Tetrabenazine – Huntington’s Disease

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  Immunology & Inflammatory Rare Diseases

  Tocilizumab – Castleman’s Disease