Therapy of Duchenne muscular dystrophy
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