Neuromuscular Disease

The neuromuscular disease group, refers to a variety of disorders[1], e.g., Duchenne’s MD (DMD), Becker’s MD (BMD), Emery-Dreifuss MD, Limb-Girdle MD, Facioscapulohumeral MD, Steinert disease, Amyotrophic Lateral Sclerosis, etc.

All these mostly genetic diseases affect the skeletal muscles and often also the heart muscle. The symptoms include muscle weakness and progressive muscle wasting and onset varies from less than 4 years of age (in the DMD) to between 20 to 70 years of age (in the BMD). Each of these diseases has an incidence rate that varies from 1 to 30 in 100,000, and some of these affect primarily males (i.e., about 1 in every 3,000 boys worldwide are born with the most common form of the disease). Given these facts, the participants are expected to be primarily male (particularly in the DMD group) and the mean age of the population in the pilot trials to be a few decades younger than the PD group.