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Now we are beginning to see clear examples of how revolutionary gene therapy can be. Most recently, a treatment for Duchenne muscular dystrophy, DMD, has been successful.
Nine boys who suffered from this serious muscle disease received gene treatment and for most of them the results are very promising. One of the boys in the study could not take more than four steps up a flight of stairs without having to stop and rest. After the treatment, he can now run up the entire stairs.
The treatment still has some side effects where the boys sometimes got a fever or problems with the kidneys. However, the researchers believe they have found a solution to the problems and it is now being tested.