Positive results from a study of ivacaftor (Kalydeco®) in children ages 1 to 2 were announced today.
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Two Phase 3 clinical trials of tezacaftor (VX-661) in combination with ivacaftor (Kalydeco®) showed positive results, Vertex Pharmaceuticals announced today.
La fibrosis quística (FQ) es una enfermedad genética. Escuche las presentaciones de los expertos, el Doctor Fadel Ruiz, director del centro de fibrosis quística de Baylor y el Doctor Carlos Milla, director del centro de fibrosis quística de Stanford y sus respuestas a las principales preguntas de la comunidad sobre terapias genéticas para la FQ. El panel fue moderado por Sylvia Mazuera, madre de dos hijos, el menor de los cuales tiene FQ.
Today, Vertex Pharmaceuticals Inc. announced that the U.S. Food and Drug Administration has accepted its application to expand Trikafta® (elexacaftor/tezacaftor/ivacaftor) to include children ages 6-11 years old with cystic fibrosis who have at least one F508del or a mutation in the CFTR gene that is responsive based on in vitro data. The FDA has granted priority review of the application and has indicated that it will make a decision by June 8, 2021.
The U.S. Food and Drug Administration (FDA) announced that it has approved Ultresa™ delayed-release capsules, a pancreatic enzyme replacement therapy. Ultresa (pancrelipase) is manufactured by Aptalis Pharma.