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Typically, when FDA approves an expansion of the indication for a drug, it means that additional clinical data have shown the drug can safely and effectively treat patient populations other than those for which it was originally intended. But collecting additional clinical data is sometimes very difficult, especially with rare diseases. Patient populations are small and are often scattered throughout the country or the world, and therefore hard to access.
Nearly 2,000 known mutations can affect the CFTR gene, which encodes for a protein that controls the movement of salt and water in and out of the body’s cells. About 300 mutations of these mutations result in CF. Certain mutations can cause more severe disease than others.
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