What's the discovery or innovation?

Current treatments address symptoms, but research shows a new drug, vosoritide, containing a natural human protein targets the cause and stimulates bone growth.

An international trial led by the Murdoch Children’s Research Institute found children treated grew an average of 1.57 centimetres per year more than those who received placebo (no treatment), and the therapy boosted bone growth at a rate almost equivalent to that of children of the same age without the condition.

Previous trials confirmed vosoritide was safe to give to children aged five to 18 years with dwarfism.

How is it changing children’s lives?

The drug has an everyday impact on quality of life and social functioning and increases access to environments. Patients treated can now climb trees, jump rocks, and are more independent. We hope it will also improve health outcomes by reducing medical complications and surgery.

Another global trial we are leading in children aged under five may improve height, body proportion, and complications, including an increased risk of sudden death. Plans are underway for vosoritide to go on the market. It could be a daily injection until the end of puberty when growth plates in bones close to finish growing.

Dwarfism and the controversial drug trial | SBS Dateline

More information

• New drug shown to improve bone growth in children with achondroplasia in global phase III trial
• Drug boosts growth in youngsters with most common form of dwarfism, new study finds
• Bone and cartilage disorders