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Gene therapy medicine has been developed to save a single baby. Development also raises questions of research ethics and finance.It is not possible to know for sure how much it cost, but it is certainly in the billions of dollars. The business raises a number of questions: how long can personalized medicine be reduced and the resources spent on research be neglected.Millions of dollars worth of medicine that can help one baby (Photo: YouTube / Julia Vitarello) The eight-year-old Mila Makovechas a rare, fatal genetic disorder, this is the Batten's. From the age of three, his condition deteriorated. Your nervous system was spurning day after day. She used to be a mobile, talkative little girl, but she lost her sight and couldn't stand or lift her head anymore. After a few years, he had to feed through many tubes and killed 30 seizures a day. It is predicted that by adolescence he would have died - the Index reports. The interesting thing about the baby's disease is that Batten's disease usually seems to be one of the most common causes of birth defects. Timothy Yu, the Boston Children's Hospital doctor has discovered that this is because Mil's entire gene is not flawless: an important white can not be synthesized in an appropriate way.You designed ribonucleic acid (RNA) to counteract the effect of the defective gene. Parents have set up a fund to fund the drug, and a campaign has been launched on the GoFundMe community-funded website. So far, it has raised over three million dollars ($ 908 million). It is not known exactly how much the drug would cost to develop.It has been tested in cell culture and experimental animals and has been shown to be effective. This time, the American drug authority, the FDA, also allowed it to start injecting the baby into the spinal cord. In this case study, the results show that the baby still lost brain cells, failed to attack, Direction: Everyone should be given a RNA fragment that specifically outweighs the effect of the mutation it contains. According to Yu and colleagues, this method can be effective in all of the hundreds of neurodegenerative diseases. However, the side effects and risks of the procedure are not known. As it stands, it is applicable only to life-threatening nervous system disorders and only when there is no other solution. They also stated that the research team is not yet ready to help other children. Other sources, however, suggest that Yu and his staff treat two other patients in a similar fashion. which she has said so far, and is not responding to the few things that made her laugh. That is, the results may be better than what you would have experienced without treatment, but there is no healing. It also raises some questions about how to evaluate the basic efficacy and safety of a drug for a single patient. Generally, for a long time, many patients are tested for a drug before being used. In this case, however, there is no such thing.
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- How much does a sick baby's life cost?
- First they try to bleach blindness through gene editing