Some muscles, including the heart, were corrected by the therapy, and this is considered a major success because heart failure surfaces as the common cause of death
among Duchenne sufferers.
These are the muscular dystrophies (
among which are
Duchenne and Becker); motor neuron diseases (including ALS and SMA); the peripheral nerve disorders (CMT and Friedreich's ataxia); inflammatory myopathies; disorders of the neuromuscular junction; metabolic diseases of muscle as well as other myopathies.