Epidemiology of Cystic Fibrosis

Cystic Fibrosis -the most common autosomal recessive disease for individuals of European
descent
-1 in 2500 newborns are affected with the disease, consequently 1 in 25
individuals are carriers.

• life expectancy -1959 (6 months)
• life expectancy -2006 (38.6 yr)
  -based on data compiled by Cystic Fibrosis Foundation - survival depends largely on the access to health care.
• 1989- the discovery of CF gene
• 1990- scientists successfully made copies of the normal gene, and added them to the CF cells in the laboratory dishes, which corrected the defective cells.
• 1993- the first experimental gene therapy treatment was given to a patient (modified common cold virus was used as carriers or delivery vehicle-carrying the genes to CF cells in the airway)
• the first drug therapy , 1993 =Pulmozyme (mucus-thinning drug) f(x)= improved respiratory infections and lung function.
• 1995, ibuprofen was found to reduce lung inflammation in children with CF, under controlled condition and in high doses.
• in late 1997, tobramycin solution for inhalation=reformulated version of antibiotics which was found to improve lung function and reduce no of hospital stays.
• other treatment strategies to correct the protein product of the gene are currently being tested in clinical trials.
  
• what makes these drugs unique is that the researchers appear to be treating the causes of CF not just the symptoms.
• in Malaysia, approximately 16 patients were diagnosed with CF from 1997 to 2003.

references: Cystic Fibrosis Foundation and National Peadiatric Institute