‘NEW MEDICATION COULD BE A GAME CHANGER’

‘Dwarfism is a congenital condition (one we are born with) that is also known as achondroplasia. And although there is a genetic link, about 80% of cases are due to a random mutation. There are multiple effects on the body, the most common are changes in the growth of the skull, a normal length trunk, but short arms and legs. The average adult height of someone with achondroplasia is about 4ft.

Vosoritide is a new (licensed in 2021) medicine for the treatment of children with achondroplasia. It’s a once-a-day injection, and could be a game changer for children who start treatment early. Obviously, though, once your bones are fused in late teens/early adult years, this cannot be reversed, so adults cannot be treated, but this could mean the end of the condition for children in the future if diagnosed and treated early.’

✱ h3health.co.uk