Video Library

From diagnosis to therapy in Duchenne muscular dystrophy

Video, Jul 29 2020

Biochemistry Focus Webinar Series

60 minutes

In this webinar, we heard from the 2020 Centenary Award winner, Professor Dame Kay Davies from the MDUK Oxford Neuromuscular Centre. During her Award Lecture, Kay presented a summary of the current status of Duchenne Muscular Dystrophy (DMD) therapy with a particular focus on those genetic strategies which have been taken forward to the clinic.

The Biochemical Society's Centenary Award is presented annually and recognizes biochemists of distinction from any part of the world. 2020 recipient, Professor Dame Kay Davies focuses her work on the development of therapeutic strategies for DMD. Kay’s earlier work paved the way for the use of mini dystrophin gene delivery using AAV and contributed to the exon skipping approach to therapy. She also discovered the dystrophin related protein, utrophin, and demonstrated that increased levels of utrophin can compensate for the lack of dystrophin in the mdx mouse model of the disease. This has led to the identification of small molecules which increase utrophin in adult mdx muscle and prevent pathology. Kay co-founded Summit Therapeutics to translate her work into the clinic.

This webinar was chaired by Dr Augustin Amour, Adaptive Immunity Project Leader at GSK and member of the Biochemical Society's Clinical & Translational Research Theme Panel.

Share This