Rare diseases
While there is a very large number of different rare diseases, this section looks at the work of just two AMRC members, each focusing on a rare but very distressing condition.
Chronic Granulomatous Disorder Research Trust
The trial was positive, the impact of gene therapy remarkable: it extended or even saved life and cleared otherwise incurable infections
British Sjögren’s Syndrome Association
A nice illustration of the way in which challenging accepted dogma can turn around understanding of a condition and suggest new ways to prevent or treat it
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