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People with diseases caused by rare mutations have fewer options and poorer prospects than other patients despite rapid recent advancements in the growing field of genetic science. In cystic fibrosis, for example, medications can vastly improve health for 90% of people with the condition, which is the most common fatal genetic disease in the U.S. But the therapies don’t work for the 10% of patients with rare mutations. Experts say one big reason for this dynamic is that it’s less profitable to develop therapies for smaller numbers of people. With help from charities, however, scientists are working on gene therapies that can help all patients, regardless of mutation.
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