Drugs that target the underlying defect in the cystic fibrosis transmembrane conductance regulator (
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Managing cystic fibrosis can feel overwhelming at times — but we’re here to help. Learn how to navigate life with CF.
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.
Anxiety is a normal emotion that comes and goes in response to fears or worries about changes in health, work, relationships, or money. A person is said to have an anxiety disorder if the anxiety does not go away, gets worse over time, and prevents a person from participating in daily activities.
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.