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Results for tag: "Familial Amyloid Neuropathies Drug"

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Familial Amyloid Neuropathies - Pipeline Review, H1 2020

  • Drug Pipelines
  • June 2020
  • 98 Pages
  • Global
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  • 1 Results (Page 1 of 1)
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Familial Amyloid Neuropathies (FAN) is a group of rare, inherited, progressive neurological disorders caused by the accumulation of amyloid proteins in the peripheral nervous system. Symptoms of FAN include sensory disturbances, muscle weakness, autonomic dysfunction, and neuropathy. Treatment of FAN is focused on slowing the progression of the disease and managing symptoms. Drugs used to treat FAN include transthyretin stabilizers, such as tafamidis, and transthyretin production inhibitors, such as patisiran. These drugs are used to reduce the production of amyloid proteins and stabilize the transthyretin protein. Other treatments include enzyme replacement therapy and gene therapy. Companies in the FAN drug market include Pfizer, Alnylam Pharmaceuticals, Ionis Pharmaceuticals, and Sanofi. Show Less Read more