University of Oxford | United Kingdom
EMBO 1991 | SciSocC 99–00
My major research effort is focussed on the development of an effective treatment of the muscle wasting disease Duchenne muscular dystrophy. We are trying to increase the levels of a protein, utrophin, which is very similar to the missing protein dystrophin. This strategy works well in mouse models and we are just starting Phase 1b trials in DMD patients.
Keywords: Muscle disease / ataxia / motor neuron disease / synapse / muscular dystrophy