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Drugs that target the underlying defect in the cystic fibrosis transmembrane conductance regulator (
Once your
Choosing to volunteer in a
Clinical trials that test potential drugs and therapies in people with cystic fibrosis are a major part of CF research. They take place at Cystic Fibrosis Foundation-accredited care centers all over the United States and enroll people with CF of all ages.
When you are deciding whether to join a clinical trial, you should consider logistics.
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.
There are two main types of clinical studies in CF research. Both are important to move research forward and provide the best outcomes for people with CF.
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.
Clinical trials are critical to developing new treatments for CF. So, how exactly does an experimental drug become an approved therapy?