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The results of the Phase 2 trial into the drug, Vosoritide, that could reduce the health complications of children with Achondroplasia was published in the New England Journal of Medicine on Tuesday 18th June 2019.

Achondroplasia is a genetic bone disorder and is the most common of the conditions causing dwarfism with around 70% of people with dwarfism having this condition. The vast majority of children born with achondroplasia, around 80%, will have average-height parents and the genetic condition will have occurred spontaneously.

People with dwarfism live normal lives; working as doctors, lawyers, accountants, teachers and shop assistants; enjoying and taking part in the same hobbies and leisure activities as the rest of society; living in families with people of average height or short stature as their partners, husbands, wives, brothers, sisters and children.

A number of children and adults, with Achondroplasia, experience health complications as a result of their condition. These include; spinal cord compression, spinal curvature, bowed legs, joint pains and problems with the inner ear.

As a support charity we are passionate about changing society for the better, so that people with dwarfism can have the same opportunities as everyone else. Our purpose is to ensure that all people who are affected by dwarfism are equal in society. We would therefore support any medical advancements that reduce the health complications of people with achondroplasia, avoid surgery, remove chronic pain and allow people to live every aspect of their lives to the fullest.

RGAUK works closely with our Medical Advisory Board who are following the research and work being carried out on Vosoritide. Doctor Melita Irving from Guy’s and St Thomas’ NHS Foundation Trust is a member of the Medical Advisory Board and conducted part of the Phase 2 trials. RGAUK supports Guy’s and St Thomas’ NHS Foundation Trust and the drug trials and are delighted that the trial has, so far, shown a potential to reduce the health complications of people with Achondroplasia.

There has been some discussion around the additional height gained as an outcome of the trial. Whilst this may help individuals when it comes to reaching things more comfortably throughout the day, RGAUK do not view this increased height as an important outcome, and if it is shown that this is the only benefit of Vosoritide and there is no reduction in the health complications, RGAUK would no longer support the development of the drug.

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