Thursday, 30 April 2020

What are monogenic diseases?? Cystic fibrous

Cystic fibrosis
Cystic Fibrosis is a genetic disorder that affects the respiratory, digestive and reproductive systems involving the production of abnormally thick mucus linings in the lungs and can lead to fatal lung infections. The disease can also result in various obstructions of the pancreas, hindering digestion. An individual must inherit two defective cystic fibrosis genes, one from each parent, to have the disease. Each time two carriers of the disease conceive, there is a 25 percent chance of passing cystic fibrosis to their children ; a 50 percent chance that the child will be a carrier of the cystic fibrosis gene; and a 25 percent chance that the child will be a non-carrier.
Prevalence:
The incidence of CF varies across the globe. Although it is severely underdiagnosed in Asia, existing evidence indicates that the prevelance of CF is rare. In the European Union 1 in 2000-3000 new borns is found to be affected by CF . In the United States of America the incidence of CF is reported to be 1 in every 3500 births.
Diagnosis/ prognosis:
People with CF have a variety of symptoms including: very salty-tasting skin; persistent coughing, at times with phlegm; wheezing or shortness of breath; an excessive appetite but poor weight gain; and greasy, bulky stools. Symptoms vary from person to person, in part, due to the more than 1,000 mutations of the CF gene, several of which have been identified and sequenced by researchers.The sweat test is the standard diagnostic test for CF. This simple and painless procedure measures the amount of salt in the sweat. A high salt level indicates CF. Although the results of this test are valid any time after a baby is 24 hours old, collecting a large enough sweat sample from a baby younger than 3 or 4 weeks old may be difficult. The sweat test can also confirm the diagnosis in older children and adults. If pancreatic enzyme levels are reduced, an analysis of the person's stool may reveal decreased or absent levels of the digestive enzymes (trypsin and chymotrypsin) or high levels of fat. If insulin secretion is reduced, blood sugar levels are high. Pulmonary function tests may show that breathing is compromised. Also, a chest x-ray may suggest the diagnosis. Relatives other than the parents of a child with cystic fibrosis may want to know if they're likely to have children with the disease. Genetic testing on a small blood sample can help determine who has a defective cystic fibrosis gene. Unless both parents have at least one such gene, their children will not have cystic fibrosis. If both parents carry a defective cystic fibrosis gene, each pregnancy has a 25 percent chance of producing a child with cystic fibrosis. During pregnancy, an accurate diagnosis of cystic fibrosis in the fetus is usually possible.
The severity of cystic fibrosis varies greatly from person to person regardless of age; the severity is determined largely by how much the lungs are affected. However, deterioration is inevitable, leading to debility and eventually death. Nonetheless, the outlook has improved steadily over the past 25 years, mainly because treatments can now postpone some of the changes that occur in the lungs. Half of the people with cystic fibrosis live longer than 28 years. Long-term survival is somewhat better in males, people who don't have pancreatic problems, and people whose initial symptoms are restricted to the digestive system. Despite their many problems, people with cystic fibrosis usually attend school or work until shortly before death. Gene therapy holds great promise for treating cystic fibrosis.
According to the CF Foundation's National Patient Registry, the median age of survival for a person with CF is currently 33.4 years. Only thirty years ago, a CF patient was not expected to reach adulthood. Many people even live into their fifties and sixties.
As more advances have been made in the treatment of CF, the number of adults with CF has steadily grown. Today, nearly 40 percent of the CF population is age 18 and older. Adults, however, may experience additional health challenges including CF-related diabetes and osteoporosis. CF also can cause reproductive problems - more than 95 percent of men with CF are sterile. But, with new technologies, some are becoming fathers. Although many women with CF are able to conceive, limited lung function and other health factors may make it difficult to carry a child to term.

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