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Drug Boosts Growth In Youngsters With Most Common Form Of Dwarfism

June 19, 2019

A ground breaking drug that helps regulate bone development has boosted growth rates in children with achondroplasia – the most common type of dwarfism – in a global trial led by Melbourne’s Murdoch Children’s Research Institute. Results of the Phase 2 trial in children aged five to 14 years are published today (Tuesday, June 18) in the New England Journal of Medicine. The project is now in Phase 3 to test the drug, vosoritide, in a larger group of patients aged 5 to 18 years. Lead author and MCRI clinical geneticist Professor Ravi Savarirayan, said achondroplasia is caused by overactivity of a signal that stops growth, and could be likened to overwatering a plant.