Based in Lexington, Mass., the Cystic Fibrosis Foundation Therapeutics Lab conducts groundbreaking CF research to help rapidly advance new therapies.
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Cystic fibrosis occurs when the cystic fibrosis transmembrane conductance regulator (CFTR) protein is either not made correctly, or not made at all. By understanding how the protein is made, scientists have been able to develop treatments that target the protein and restore its function.
Two-thirds of people with cystic fibrosis in the U.S. experience debt, food insecurity, or housing issues.
The journey to end cystic fibrosis isn't a straight line. It is an evolving map with many paths and unique challenges. It requires an ambitious research agenda to accelerate treatments and drug development for the underlying cause of the disease and ultimately deliver a cure.
Results show that people on Trikafta® enrolled in the six-week study were able to safely stop taking one of the two common CF medications without negatively affecting their health.
The Foundation and Bakar Labs will support AVECRIS Pte Ltd and Nosis Biological Sciences as they pursue genetic therapies for cystic fibrosis with their novel technologies.
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.
The Cystic Fibrosis Foundation participated in a Congressional briefing hosted by Friends of Cancer Research on July 24 to educate members of the U.S. Senate about new processes developed by the FDA to speed the approval of breakthrough therapies.
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