Narrative
New therapies supported by clear evidence from clinical trials have resulted in outstanding improvements in survival and quality of life for people living with cystic fibrosis (CF). Elborn’s clinical trials group has delivered a programme of crucial clinical trials which has impacted on clinical practice in CF. From 2009-2012 Elborn co-led a pivotal multicentre trial using Ivacaftor (Kalydeco TM), a transformative new drug which represents a paradigm shift as the first approved therapy that corrects the basic defect in CF. This therapy is an exemplar of personalised medicine and is prescribed for patients with the specific gene mutation in which this drug works.| Impact status | Ongoing |
|---|---|
| Category of impact | Health Impact, Quality of Life Impact |
Related content
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Research output
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Guideline on the design and conduct of cystic fibrosis clinical trials: The European Cystic Fibrosis Society–Clinical Trials Network (ECFS-CTN)
Research output: Contribution to journal › Literature review › peer-review
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Clinical trials in cystic fibrosis
Research output: Contribution to journal › Literature review › peer-review
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Elective versus symptomatic antibiotic treatment in cystic fibrosis patients with chronic Pseudomonas infection of the lungs
Research output: Contribution to journal › Article › peer-review
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A CFTR Potentiator in Patients with Cystic Fibrosis and the G551D Mutation
Research output: Contribution to journal › Article › peer-review
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The effect of treatment of cystic fibrosis pulmonary exacerbations on airways and systemic inflammation
Research output: Contribution to journal › Article › peer-review
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Safety and efficacy of recombinant alpha(1)-antitrypsin therapy in cystic fibrosis.
Research output: Contribution to journal › Article › peer-review
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Press/Media
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The challenges of end of life care in cystic fibrosis
Press/Media: Public Engagement Activities