Patient Registry for Bardet-Biedl Syndrome
We’re working with UCL to find someone to help develop a patient registry for BBS UCL are setting up a professional registry for people in the UK with the rare genetic condition […]
rare genetic condition
New Treatment for Spinal Muscular Atrophy
written by Dr Thomas Dunne What is Spinal Muscular Atrophy? Spinal Muscular Atrophy (SMA) is a rare genetic condition that causes progressive weakness and wasting of the muscles. It is […]