Efficacy of Ivacaftor in a Child with Cystic Fibrosis, End-Stage Lung Disease, and Cepacia Syndrome
| Autor: | Peter Hiatt, Shipra Singh, Christopher M. Oermann, Walter A. Castro-Elias, Robert H. Moore |
|---|---|
| Rok vydání: | 2012 |
| Předmět: |
Pulmonary and Respiratory Medicine
congenital hereditary and neonatal diseases and abnormalities medicine.medical_specialty business.industry Potentiator medicine.disease Cystic fibrosis Ivacaftor Clinical trial Lung disease Internal medicine Pediatrics Perinatology and Child Health medicine Immunology and Allergy Stage (cooking) Intensive care medicine business medicine.drug |
| Zdroj: | Pediatric Allergy, Immunology, and Pulmonology. 25:231-233 |
| ISSN: | 2151-3228 2151-321X |
| Popis: | Ivacaftor is a small-molecule potentiator of the cystic fibrosis (CF) transmembrane-conductance regulator and was recently approved for use in CF patients with at least one copy of the G551D mutation. Two clinical trials have proven its efficacy in CF patients 6 years of age and older with mild-to-moderate lung disease. In this article, we report the efficacy of ivacaftor in a 12-year-old CF patient with very severe lung disease and clinical features of cepacia syndrome. |
| Databáze: | OpenAIRE |
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