Inadequate Treatment of Duchenne Muscular Dystrophy Patients


Having cared for many hundreds of boys with Duchenne muscular dystrophy, it was encouraging to see an article by Nicholas Wade in the January 1, 2016 New York Times. Three researchers are trying a new technique to insert "the correct gene into damaged cells."

Over the years, there have been countless drug and other research trials,  but this offers a new approach. Scientifically, the article was excellent, but it contained several statements that were extremely bothersome. In the second paragraph, the writer stated that the disorder puts boys in wheelchairs by age ten. This should never happen if the patients are under the care of physicians with neuromuscular training. Unfortunately, in the U.S. we have few adequate clinics or programs for these patients. Other countries are far ahead of us in their treatment of patients with Duchenne dystrophy.

I have had youths walk until age 15 and they did not have the more benign form of Becker muscular dystrophy. Right from the start, parents should be taught to stretch the boy's  heelcords, then night braces and heel cord lengthening at the proper time all make a difference. Also, prednisone given in appropriate doses and carefully monitored can help greatly. However, if a boy is not seen at least every two weeks or so by a physician and both weight and blood pressure monitored, problems can result. A low salt diet while on the prednisone is also important. I saw one patient who was on high doses of prednisone and had gained 50 pounds in a short time. The arrogant neurologist had not seen him for six months! I was appalled!

I was on a book tour for my first book, Raising a Handicapped Child, and was looking forward to meeting a former patient and his lovely parents in a hospital clinic in a northern state. A clinic for Duchenne dystrophy patients was in progress in an adjoining room and I was greatly concerned to see the little boys walking on their toes. I asked the nurse about this and she said their orthopedists did not seem to know about heelcord lenthenings. Sadly, this is quite common in the U.S. because neuromuscular training is seldom given to orthopedic surgeons.

I was married to an orthopedic surgeon and saw what training he received in one of the most outstanding orthopedic programs. Heelcord lengthenings, short leg braces, and night braces can make a real difference. Also, after surgery a youth should be up and walking as soon as possible. Physical therapy can make a great difference in retaining the ability to walk. Dr. Paul Vignos, who first advocated this form of treatment, taught me a great deal about caring for these youths. He was not an orthopedist, but worked with one and published some  important articles about how well these boys can do if they receive the treatment he advocated.


Image copyright: Jens karlsson/flickr/CC BY

Article last time updated on 05.08.2016.

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