Kalydeco Marks the Latest Success of the Cystic Fibrosis Foundation’s Venture Philanthropy Model
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CF Foundation Provided Scientific, Clinical and Significant Funding Support in Drug Development Process
This medicine represents the single greatest therapeutic advancement in the history of CF, offering a treatment for the underlying cause of the disease that could eventually benefit more than 90 percent of people with CF.
Drugs that target the underlying defect in the cystic fibrosis transmembrane conductance regulator (
Drug Targets the Underlying Cause of the Disease in People with the Most Common CF Mutation
Every person has two copies of the cystic fibrosis transmembrane conductance regulator (CFTR) gene. A person must inherit two copies of the CFTR gene that contain mutations — one copy from each parent — to have cystic fibrosis.
Estamos buscando tratamientos potenciales para las personas con FQ que tienen mutaciones raras y sin sentido. Es posible que no aprovechen de los moduladores, que corrigen la proteína reguladora de la conductancia transmembrana de la fibrosis quística (CFTR, por sus siglas en inglés) que funciona mal.