Here's what you need to know about the four CFTR (cystic fibrosis transmembrane conductance regulator) modulators.
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Drugs that target the underlying defect in the cystic fibrosis transmembrane conductance regulator (
Theratyping matches therapies, or medications, to specific types of mutations. The primary goal of theratyping is to identify which mutations respond to certain CFTR modulators, thereby helping people with rare CFTR mutations gain access to already approved modulators quickly and safely.
Caring for anyone with a long-term disease — especially if that person is your child — is stressful. Parents of children with cystic fibrosis are more likely to experience anxiety, depression, or both compared to parents in general.
People with chronic diseases, such as cystic fibrosis, are at greater risk for developing clinical depression. When left untreated, depression can interfere with your ability to manage your CF effectively and experience a better quality of life.
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.