Nick is the parent of a son, Saul, who is living with Duchenne muscular dystrophy. He was the founder member and former CEO for the charity, Action Duchenne. Nick has an honours degree in Chemistry and a PGCE. He established the first patient clinical trial database, the DMD Registry, and is currently a member of its advisory board. He is also a member of the Treat NMD TACT committee for evaluating new treatments and medicines for Duchenne. From 2005 until 2012, he was a member of the MDEX Consortium SAB, which supported the first clinical trial for exon skipping using morpholinos with boys living with DMD in the UK. When CEO at Action Duchenne, Nick established and contributed to the first of many Duchenne International Research Conferences in the UK and he also set up the APPG for Muscular Dystrophy in the Houses of Parliament. He has recently campaigned with families to win approval for the PTC genetic therapy Translarna with NICE and NHS England.
Published on 21 August 2017Share this article Categories Uncategorised