New and investigational treatments in cystic fibrosis
Cystic fibrosis (CF) is an autosomal recessive disorder that affects approximately 1 in 3000 Caucasian births, or 30,000 individuals in the US and 70,000 worldwide. The discovery of the CF gene, isolation of the CFTR protein and understanding of molecular mechanisms behind the clinical expression of...
| Main Authors: | , |
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| Format: | Article |
| Language: | English |
| Published: |
SAGE Publishing
2011-08-01
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| Series: | Therapeutic Advances in Respiratory Disease |
| Online Access: | https://doi.org/10.1177/1753465811398267 |
| _version_ | 1811330804076249088 |
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| author | Mangala Narasimhan Rubin Cohen |
| author_facet | Mangala Narasimhan Rubin Cohen |
| author_sort | Mangala Narasimhan |
| collection | DOAJ |
| description | Cystic fibrosis (CF) is an autosomal recessive disorder that affects approximately 1 in 3000 Caucasian births, or 30,000 individuals in the US and 70,000 worldwide. The discovery of the CF gene, isolation of the CFTR protein and understanding of molecular mechanisms behind the clinical expression of CF are being translated into newer treatments. Treatments for CF and its manifestations are discussed in this article including inhaled antibiotics, hydrator therapies, anti-inflammatory agents and protein modifiers. New and experimental treatments that are in development are also discussed. Outcomes for these treatments are forced expiratory volume in one second (FEV 1 ) improvement, CF-related quality of life, use of intravenous antibiotics and frequency of exacerbations and hospitalizations. |
| first_indexed | 2024-04-13T16:08:31Z |
| format | Article |
| id | doaj.art-e7daa40e56d14da2959230a1f4044c20 |
| institution | Directory Open Access Journal |
| issn | 1753-4658 1753-4666 |
| language | English |
| last_indexed | 2024-04-13T16:08:31Z |
| publishDate | 2011-08-01 |
| publisher | SAGE Publishing |
| record_format | Article |
| series | Therapeutic Advances in Respiratory Disease |
| spelling | doaj.art-e7daa40e56d14da2959230a1f4044c202022-12-22T02:40:20ZengSAGE PublishingTherapeutic Advances in Respiratory Disease1753-46581753-46662011-08-01510.1177/1753465811398267New and investigational treatments in cystic fibrosisMangala NarasimhanRubin CohenCystic fibrosis (CF) is an autosomal recessive disorder that affects approximately 1 in 3000 Caucasian births, or 30,000 individuals in the US and 70,000 worldwide. The discovery of the CF gene, isolation of the CFTR protein and understanding of molecular mechanisms behind the clinical expression of CF are being translated into newer treatments. Treatments for CF and its manifestations are discussed in this article including inhaled antibiotics, hydrator therapies, anti-inflammatory agents and protein modifiers. New and experimental treatments that are in development are also discussed. Outcomes for these treatments are forced expiratory volume in one second (FEV 1 ) improvement, CF-related quality of life, use of intravenous antibiotics and frequency of exacerbations and hospitalizations.https://doi.org/10.1177/1753465811398267 |
| spellingShingle | Mangala Narasimhan Rubin Cohen New and investigational treatments in cystic fibrosis Therapeutic Advances in Respiratory Disease |
| title | New and investigational treatments in cystic fibrosis |
| title_full | New and investigational treatments in cystic fibrosis |
| title_fullStr | New and investigational treatments in cystic fibrosis |
| title_full_unstemmed | New and investigational treatments in cystic fibrosis |
| title_short | New and investigational treatments in cystic fibrosis |
| title_sort | new and investigational treatments in cystic fibrosis |
| url | https://doi.org/10.1177/1753465811398267 |
| work_keys_str_mv | AT mangalanarasimhan newandinvestigationaltreatmentsincysticfibrosis AT rubincohen newandinvestigationaltreatmentsincysticfibrosis |