New drugs are expected to increase the height of children with dwarfism

New drugs are expected to increase the height of children with dwarfism

New drugs are expected to increase the height of children with dwarfism

June 26, 2019 Source: Chinese Journal of Science and Technology

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According to an international clinical trial led by Xinhua News Agency's Murdoch Children's Institute in Australia, a drug that helps regulate bone development can significantly increase the growth rate of children with achondroplasia, which is expected to make them grow taller.

Cartilage hypoplasia is the most common type of dwarfism caused by genetic mutations. The patient's stature is short and bone growth is disproportionate, often accompanied by complications such as spinal cord compression, spinal curvature, and arched legs.

The researchers said that achondroplasia is caused by a signal that prevents growth from being overactive, while the drug Vosoritide inhibits this signal and promotes normal bone development. To further understand the safety, tolerability, and optimal dosage of this drug, the researchers selected 35 children aged 5 to 14 years to conduct clinical trials.

In trials with a total duration of more than 4 years, the children were divided into groups and received subcutaneous injections every day during the first year, but the doses were different.

The results showed that the average growth rate of the subjects increased by 50%, and the children who received the specific dose of the drug had the fastest growth rate and could continue for several years after stopping the drug. Adverse reactions occurred in all subjects. But according to the researchers, most of the adverse drug reactions are "moderate."

The above research results have been published in the new issue of the New England Journal of Medicine. The researchers said that it is hoped that this study will help improve the health of children with achondroplasia and increase their height. Larger clinical trials are currently underway . (Guo Yang)

Chinese Journal of Science (2019-06-25 2nd Edition International)

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