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4th Quarter 2019

Key topics in this quarter’s edition include:

  • Trikafta (elexacaftor/ tezacaftor/ ivacaftor; ivacaftor) for treatment of cystic fibrosis in patients who have at least one F508del mutation in the CFTR gene
  • Adakveo® (crizanlizumab) for reduction of the frequency of vasoocclusive crises in patients with sickle cell disease
  • Voxelotor for treatment of hemolytic anemia in patients with sickle cell disease
  • AR-101 to reduce the incidence and severity of allergic reactions after accidental exposure to peanut in patients with peanut allergy

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