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ATTR

ATTR
Developing small molecule stabilisers to stabilise transthyretin in systemic transthyretin amyloidosis (ATTR)

Prof Sir Mark Pepys and his team at the Wolfson Drug Discovery Unit, UCL Centre for Amyloidosis and Acute Phase Proteins have identified a novel pathway leading to the progression of transthyretin amyloidosis, enabling the development of new therapeutics. This project is funding further analysis of the pathway for formation of amyloid deposits in this disease and identification of modulators of this pathway which will lead to the development of novel and patentable chemical entities to take forward into the clinic.