The new drug is expected to make children with dwarfism grow taller

an international clinical trial led by the Murdoch Children's Institute in Australia has shown that a drug that helps regulate bone development can significantly increase the growth rate of children with cartilage dysplocy, which is expected to make them grow taller.
cartilage development is the most common type of dwarfism, caused by genetic mutations, patients are small and bone growth is disproportionate, often accompanied by spinal cord pressure, spinal bending, bow legs and other complications.
researchers say cartilage dysplase is caused entirely by overactiveness of a signal that prevents growth, and the drug Vosoritide suppresses this signal and promotes bone development to return to normal. To learn more about the drug's safety, tolerance and optimal dosing, the researchers selected 35 children between the ages of 5 and 14 and conducted clinical trials.
in trials with a total length of more than 4 years, children were divided into groups and treated with subsuplisal injections daily through the first year, but the dosing of the drug was different.
results showed that the average growth rate of the subjects increased by 50 per cent, with children receiving a specific dose of the drug growing the fastest and sustained for several years after the drug was stopped. All subjects experienced adverse reactions. But according to the researchers, adverse drug reactions are mostly "moderate."
the findings have been published in the new issue of the New England Journal of Medicine. The researchers say they hope the study will help improve the health of children with cartilage development and increase their height. Larger clinical trials are already under way. (Source: Xinhua News Agency, Guo yang)
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