Researchers continue to investigate stem cells, gene therapy, and bone marrow transplants as treatments for many different genetic disorders. While genetic disorders aren't curable yet, there are other medications that can help provide relief from symptoms and improve life expectancy in some disorders, such as:
Sickle cell anemia: "Young children are recommended to take antibiotics on a regular basis to prevent infections, plus pain medication," says Yutaka Niihara, M.D., a professor of hematology at the University of California, Los Angeles School of Medicine. One of the most promising medications is hydroxyurea, which reduces the frequency of pain and other complications, and likely improves life expectancy. Avoiding cold weather, high altitudes, and dehydration can also help avoid sickle cell pain and other symptoms.
Cystic fibrosis: Children with CF are often treated with antibiotics to prevent infection, mucus-thinners, ibuprofen to reduce lung inflammation, and medicines to open up the air passages in the lungs. Kids with CF often also need to take extra vitamins or medications to help them properly digest food. If lungs become irreversibly damaged over time, a lung transplant can help people live longer and with fewer symptoms.
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