Research Approaches towards a Cure for Duchenne Muscular Dystrophy

This report has been written for families who have one or more boys with Duchenne muscular dystrophy. It explains some basic scientific facts and shows the now numerous approaches with which research is trying to find a scientifically justified and thus effective therapy of Duchenne muscular dystrophy. Because scientists in more than one hundred laboratories in many countries of the world are working to find a cure for this disease, only the most important of their results are described here in greater detail. Some further results are reported in a strongly abbreviated fashion. The first edition of this report in 2001 was based on an international workshop on Duchenne therapy research in May 2000 at the National Institutes of Health in Bethesda near Washington. This third edition of the report was written in July and August 2003 mostly with information from the literature and by correspondence with many of the researchers.