Drug to boost growth in children with dwarfism shows promise


A drug that helps regulate bone development has increased growth rates in children with achondroplasia. Photo: Pexels

A drug that helps regulate bone development has been found to increase growth rates in achondroplasia children, the most common type of dwarfism, in a global study.

The average increase in patient height to about 6 cm per year was close to the growth rates among middle-aged children, and the side effects of the drug were mostly mild, said Julie Hoover, assistant professor at the University of Washington . the McKusick-Nathan Institute of Genetic Medicine, Johns Hopkins University.

"At this time, the study results show an impact on growth, and this effect is sustained, at least for almost four years at this trial," Hoover said.

The results of the phase 2 study published in the New England Journal of Medicine showed that the drug, vosoritide, was generally well tolerated by patients.

On average, study participants grew 50% faster compared to the reference period, with no adverse effects on body proportion, the results showed.

Achondroplasia is caused by too much activity from a signal that disrupts growth, and can be compared to an excess of water in a plant, said author Ravi Savarirayan, a professor at the Murdoch Children's Research Institute in Australia.

"This drug basically twists the hose so the plant gets the right amount of water and can regain regular growth," said Savarirayan.

Achondroplasia is a genetic bone disorder that affects about one in 25,000 babies.

It is caused by a mutation in the FGFR3 gene that impairs bone growth in the limbs, spine and base of the skull.

The most common health complications in children with achondroplasia are compression of the spinal cord, curvature of the spine, and arched legs. About half of these children will need spinal or other surgery.

Unlike other treatments – such as growth hormone and limb stretching surgery – that focus on symptoms, vosoritide focuses on the underlying cause of achondroplasia and directly neutralizes the effect of the growth-retarding mutation.

The study lasted four years at research centers in Australia, France, Britain and the USA, with 35 children assigned to one of the four groups who received daily subcutaneous doses of the drug in increasing amounts.

The results showed that vosoritide demonstrated dose-dependent increases in centimeters grown per year during the first six months, with improvements maintained over the extended study period of another three years.



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