Therapy Areas: Hereditary Disorders
8 May 2019 - - The US Food and Drug Administration granted Fast Track designation for Molgradex, an inhaled formulation of recombinant human granulocyte-macrophage colony-stimulating factor (GM-CSF), ...
2 May 2019 - The United States Food and Drug Administration (FDA) has approved United States-based Vertex Pharmaceuticals Incorporated's (Nasdaq:VRTX) KALYDECO (ivacaftor) intended for use in chi...
11 April 2019 - Biopharmaceutical company Synspira Therapeutics reported on Wednesday the receipt of Orphan Drug Designation (ODD) from the European Medicines Agency (EMA) Committee for Orphan Medicin...
12 March 2019 - - The Therapeutic Goods Administration of Australia has granted registration to Symdeko (tezacaftor/ivacaftor and ivacaftor) for the treatment of people with cystic fibrosis aged 12 ye...
8 March 2019 - - Treatment with the triple combination of the next-generation corrector VX-445, tezacaftor and ivacaftor resulted in statistically significant improvements in lung function (percent pr...
20 February 2019 - - US-based biotechnology company Vertex Pharmaceuticals Inc. (NASDAQ: VRTX) has received the results of a Phase 3 study conducted in Europe and Australia of tezacaftor in combi...
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