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The Cystic Fibrosis Trust (stylised as Cystic Fibrosis) is a UK-based national charity founded in 1964, dealing with all aspects of cystic fibrosis (CF). It funds research to treat and cure CF and aims to ensure appropriate clinical care and support for people with cystic fibrosis.
In December 2020, after an additional clinical trial was completed, and FDA approval was expanded for 177 other cystic fibrosis mutations. [32] FDA approval for children aged 6–11 was added in January 2021, after a third clinical trial was completed. [33] In 2023, approval was extended to children 2–5. [34]
The EVOLVE trial analyzed tezacaftor/ivacaftor in participants with cystic fibrosis, specifically with the homozygous for Phe508del mutation. [18] The EVOLVE trial is a phase 3, double-blinded, multicenter, randomized, placebo-controlled, parallel-group trial, that was which evaluated therapy with a combination of tezacaftor and ivacaftor in participants that are aged 12 years of age and older.
Clinical trial number NCT05076149 for "A Study of VX-121 Combination Therapy in Participants With Cystic Fibrosis (CF) Who Are Homozygous for F508del, Heterozygous for F508del and a Gating (F/G) or Residual Function (F/RF) Mutation, or Have At Least 1 Other Triple Combination Responsive (TCR) CFTR Mutation and No F508del Mutation" at ...
Vertex Pharmaceuticals (NASDAQ: VRTX) fell by 13% on Dec. 19, when the rare-disease drug developer reported some lackluster results from a late-stage clinical trial. But far from being a reason to ...
The Foundation was established in 1955 by a group of volunteers in Philadelphia, Pennsylvania.. In addition to providing grants for research into cystic fibrosis and supporting clinical trials, the foundation promotes and accredits 115 specialized centers for treatment of individuals with cystic fibrosis.
Vertex achieved tremendous success with its cystic fibrosis (CF) therapies. ... and some people in the clinical trial took it less frequently than once per month; it's possible that greater weight ...
A third clinical trial examined the efficacy of ivacaftor in people with cystic fibrosis due to G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N, or S549R mutations. This trial, which included 39 people of age greater than six years, used a crossover design. The people in the trial had FEV1 averaging 78% of normal at baseline.
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