Scientists are developing new drugs that could help children with dwarfism grow taller
Last Update: 2020-06-19
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Achondroplasia is the most common type of dwarfism, which is caused by gene mutationThe patient is short and the growth of bone is not in proportion, often accompanied by complications such as spinal cord compression, spinal curvature, arched legs and so on< br / > according to the researchers, achondroplasia is caused by an overactive signal that blocks growth, and the drug vosoritide can inhibit this signal and promote bone development to return to normalIn order to further understand the safety, tolerance and optimal dosage of the drug, the researchers selected 35 children aged 5 to 14 years old for clinical trials< br / > in the trial with a total length of more than 4 years, the children were divided into several groupsIn the first year, they received subcutaneous injection every day, but the dosage was differentThe results showed that the average growth rate of the subjects increased by 50%, and the growth rate of the children who received specific doses of drugs was the fastest, and the growth rate was still several years after the drug was stoppedAll subjects had adverse reactionsBut according to the researchers, adverse drug reactions are mostly "mild."< br / > the above results have been published in the New England Journal of medicineThe researchers say they hope the study will help improve the health and height of children with achondroplasiaAt present, larger clinical trials have been launched
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