Site Search
mRNA therapy is one way to deliver the correct genetic instructions to cells, which would allow them to make functional CFTR protein regardless of an individual’s CF mutations.
Las personas con fibrosis quística y sus familias se han enfrentado a importantes cargas económicas debido a los programas acumuladores de copago. Familiarícese con estos programas y lo que implican para usted y sus seres queridos.
People with cystic fibrosis and their families have faced significant cost burdens due to copay accumulator programs. Get caught up on these programs and what they mean for you and your loved ones.
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.
Cystic fibrosis is caused by mutations in both copies of the cystic fibrosis transmembrane conductance regulator (CFTR) gene. Scientists are examining whether it is possible to correct the mutations through a process called gene editing.
With promising new discoveries in the pipeline and advancements we've made in the search for new treatments and a cure, we must continue our work to protect access to adequate, affordable insurance that preserves access to high-quality cystic fibrosis care; and support progress toward new therapies and a cure.
Robust funding for the National Institutes of Health helps ensure innovation in basic research and a full pipeline of cystic fibrosis therapies. A well-resourced U.S. Food and Drug Administration helps advance therapies that are safe and effective.