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Drug To Boost Growth In Kids With Dwarfism Shows Promise

Drug To Boost Growth In Kids With Dwarfism Shows Promise

Sentinel Digital DeskBy : Sentinel Digital Desk

  |  20 Jun 2019 10:23 AM GMT

Melbourne: A drug that helps regulate bone development has been found to boost growth rates in children with achondroplasia, the most common type of dwarfism, in a global trial. The patients’ average boost in height to about 6 cm (2.4 inches) per year was close to growth rates among children of average stature, and the side effects of the drug were mostly mild, said study co-author Julie Hoover, Associate Professor at the McKusick-Nathans Institute of Genetic Medicine, Johns Hopkins University. “Right now, the results of the study show an impact on growth, and this effect is sustained, at least over nearly four years in this trial,” Hoover said.

Results of the phase-2 trial published in the New England Journal of Medicine showed that the drug, vosoritide, was generally well tolerated by patients. On average, participants in the trial grew at a 50 per cent faster compared to baseline with no adverse effects on body proportion, the results showed. Achondroplasia is caused by over-activity of a signal that stops growth, and could be likened to overwatering a plant, said lead author Ravi Savarirayan, Professor at Melbourne’s Murdoch Children’s Research Institute in Australia. Achondroplasia is a genetic bone disorder affecting about one in every 25,000 infants. (IANS)

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