Improving access to new treatments in rare diseases

NICE

21 June 2018 - Emily Crossley, co-founder and co-chief executive of Duchenne UK, tells us how a ground-breaking HERCULES project will help NICE to get the evidence we need faster.

I was working as a journalist when my son, Eli, was diagnosed with Duchenne Muscular Dystrophy (DMD). In that moment, I went from covering other people’s tragedies to being at the centre of my own.

We started looking into treatment options, only to find there were effectively none. There are many promising treatments in development, but small patient populations and limited resources mean gathering evidence is a significant challenge.

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Michael Wonder

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Michael Wonder