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The firm's four medicines are being used to treat most of the people with CF in North America, Europe and Australia. The company offers medicines for people with serious diseases with a focus on specialty markets. The company has a pipeline of investigational therapies in other serious diseases where it is leveraging insight into causal human biology, including sickle cell disease, beta thalassemia, APOL1-mediated kidney disease, type 1 diabetes, pain, alpha-1 antitrypsin deficiency, and muscular dystrophies. The firm's marketed medicines are TRIKAFTA/KAFTRIO (elexacaftor/tezacaftor/ivacaftor and ivacaftor), SYMDEKO/SYMKEVI (tezacaftor/ivacaftor and ivacaftor), ORKAMBI (lumacaftor/ivacaftor) and KALYDECO (ivacaftor).
The firm is focused on developing medicines that treat the underlying cause of cystic fibrosis (CF) and it has several ongoing clinical and research programs to advance and extend treatment of CF. The company is headquartered in Boston, Massachusetts and currently employs 3,900 full-time employees.
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