Is Duchenne muscular dystrophy a specific type of muscular dystrophy?
Dr. Hoffman: Duchenne muscular dystrophy is the most common type of neuromuscular disorder. There are about 40 different neuromuscular disorders, including things like ALS disorders, but Duchenne is generally considered the most common worldwide and also one of the more severe. How long did the study last? Dr. Hoffman: The study took about three years. One of the unique aspects of the study is how internationally and nationally collaborative it was. It involved contributions from the National Institutes of Health, six different centers in the United States, and the Japanese government, as well as many foundations including the Foundation to Eradicate Duchenne. There were many people working together in this very difficult study and very expensive study, to show that this was possible, that we could actually help these dogs and the implications for humans. Each of these dogs that we treated for four to six months cost about $200,000 to $300,000 just in drug costs. Clearly, this presents
