Limb-girdle dystrophies begin in shoulders and hips

Ask Dr. Keith Roach M.D

DEAR DR. ROACH: I have two boys diagnosed with limb girdle muscular dystrophy. I am looking everywhere for some help, but can’t seem to find anything. Is there a cure? — F.I.I. ANSWER: There are many types of muscular dystrophy. Some start in childhood; others later in life. The limb-girdle muscular dystrophies are a diverse group of inherited disorders, caused by many different genetic abnormalities. The most common of these starts in childhood. “Limb-girdle” means that the symptoms of the disease usually start in the muscles around the hips and shoulders, as opposed to the muscles of the hands and feet. The prevalence of all types of LGMD together is estimated to be about 2 in every 100,000 people. The diagnosis of childhood-onset muscular dystrophy usually starts with a parent who notices something wrong, followed by a careful history, exam and appropriate blood testing by the pediatrician. Since there are many types of muscular dystrophy, further testing and consultation with an expert are usually necessary. The exact diagnosis used to be made by muscle biopsy, but it is now most commonly made by genetic testing. Sometimes both are needed. Because the diseases within the LGMD group are so diverse, I can’t give you details on how the condition will progress in your boys. Even your two boys may have very different onset and types of symptoms. I can point you to an excellent resource with lots more information at mda.org/disease/ limb-girdle-muscular-dystrophy. They can connect you with experts near your area. Unfortunately, there is no cure yet and, as of now, no treatment that can slow down the progression of LGMD. There are some new types of treatments being researched, including gene therapy, and I sincerely hope that effective treatments are not too far away. *** DEAR DR. ROACH: My daughter was diagnosed with prediabetes. She needs to lose weight. Is Weight Watchers a good idea? — Anon. ANSWER: In my practice, we have an excellent registered dietitian nutritionist available for individual consultation, so I usually refer there for a personalized approach. However, not everyone has access to this resource. Weight Watchers (now WW) is one of many commercial weight-loss programs in the U.S. It has been studied many times, and been shown to promote more weight loss than a control group, who were given printed materials, a few visits with a doctor or health education. The weight loss was not large: 2.6% more than the control group. Other weight loss programs, including Jenny Craig and Optifast, also showed benefit. I have had many patients use Weight Watchers, and they appreciate the simplicity of use. Some people do much better than the average, and of course some people don’t do well at all. There is no single best choice for everyone. As far as your daughter’s prediabetes goes, any program that helps people to lose weight is likely to improve the prediabetes, but avoiding simple sugars and processed starches is important. Weight Watchers has one study showing it is particularly good with prediabetes, with participants losing 5.6% of body weight compared with 0.2% of the control group at one year. If she wants to try WW, it is a reasonable choice.