Developing new therapies for motor neurone disease (SMA and ALS)

Work in our laboratory is trying to identify new and effective ways to protect motor neurons (and other affected cells and tissues of the body) against disease in both childhood and adult forms of MND. A lot of our focus is currently on spinal muscular atrophy (SMA), where we are using both drug-based and gene therapy-based approaches to target key disease pathways. In particular, we are focussing on so-called ‘combinatorial therapies’ where you bring together multiple treatments in order to get maximum clinical benefit. We are also exploring how therapies developed for SMA may be applicable to help patients with ALS.