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Diseases

Genetic and Rare Diseases Information Center (GARD)

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Cystic fibrosis


Other Names for this Disease

  • CF
  • Mucoviscidosis
See Disclaimer regarding information on this site. Some links on this page may take you to organizations outside of the National Institutes of Health.

Treatment

Management Guidelines

  • GeneReviews provides current, expert-authored, peer-reviewed, full-text articles describing the application of genetic testing to the diagnosis, management, and genetic counseling of patients with specific inherited conditions. Click on the link to view the article on this topic.

Clinical Trials & Research for this Disease

  • The Childhood Liver Disease Research and Education Network (ChiLDREN) is a team of doctors, nurses, research coordinators, and research labs throughout the U.S., working together to improve the lives of people with rare liver diseases through research. Current studies are enrolling people with¬†Alagille syndrome, alpha one-antitrypsin deficiency,¬†bile acid synthesis defects, biliary atresia, cystic fibrosis liver disease, idiopathic neonatal hepatitis,¬†mitochondrial hepatopathies, and progressive familial intrahepatic cholestasis. Click on the link above to learn more.

    Visit the following link to find the participating research center nearest you.
    http://childrennetwork.org/centers.html

  • ClinicalTrials.gov lists trials that are studying or have studied Cystic fibrosis. Click on the link to go to ClinicalTrials.gov to read descriptions of these studies.
  • The Cystic Fibrosis Research Program is funded by the National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) to support investigator-initiated research grants encompassing both fundamental and clinical studies of the etiology, molecular pathogenesis, pathophysiology, diagnosis, and treatment of cystic fibrosis and its complications.

Medical Products

The medication(s) listed in the table(s) below have been approved by the Food and Drug Administration (FDA) for treatment of this condition. The FDA Office of Orphan Products Development designates "orphan products" for those that treat rare diseases affecting fewer than 200,000 Americans. The table(s) below may not be an exhaustive list of drugs or products used to treat this condition. There may be other products available that are not considered orphan products. To search for all FDA approved drugs, visit Drugs@FDA. You can find orphan products used to treat other conditions by searching the Orphan Drug Product Designation database.


Generic Name Aztreonam
Trade Name
(Manufacturer Name)
Cayston
(Gilead Sciences)
Indication
The FDA has approved this product to be used in this manner.
To improve respiratory symptoms in cystic fibrosis (CF) patients with Pseudomonas aeruginosa
More Information about this product Drug Information Portal

Generic Name Ivacaftor
Trade Name
(Manufacturer Name)
Kalydeco
(Vertex Pharmaceuticals, Inc.)
Indication
The FDA has approved this product to be used in this manner.
Treatment of cystic fibrosis (CF) in patients age 6 years and older who have a G551D mutation in the CFTR gene
More Information about this product Drug Information Portal
Medline Plus Health Information

Generic Name Dornase alfa
Trade Name
(Manufacturer Name)
Pulmozyme®
(Genentech, Inc.)
Indication
The FDA has approved this product to be used in this manner.
In conjunction with standard therapies in the management of cystic fibrosis patients to reduce the frequency of respiratory infections requiring parenteral antibiotics and to improve pulmonary function.
More Information about this product Drug Information Portal
Medline Plus Health Information

Generic Name Tobramycin for inhalation
Trade Name
(Manufacturer Name)
Tobi®
(Novartis Pharmaceuticals Corp)
Indication
The FDA has approved this product to be used in this manner.
Management of cystic fibrosis patients with P.aeruginosa.
More Information about this product Drug Information Portal
Medline Plus Health Information

Other Names for this Disease
  • CF
  • Mucoviscidosis
See Disclaimer regarding information on this site. Some links on this page may take you to organizations outside of the National Institutes of Health.