New Treatments Slow The Progression of Muscular Dystrophy, Study Says

According to a story from Medical Xpress, two new treatments were able to slow down the worsening of symptoms associated with muscular dystrophy in a mouse model study. Tamoxifen and raloxifene were the two medications that were being tested, and in the study they showed improved muscular function (including the heart and breathing muscles) as well as greater bone density.
Muscular dystrophy is a genetic muscle disease in which the skeletal muscles progressively weaken and break down over time. There are several different types of muscular dystrophy that vary by how rapidly symptoms progress, when they begin, and the degree of severity. Genetic mutations that inhibit the production of dystrophin are responsible for the muscular break down. Symptoms include muscle wasting and loss, scoliosis, inability to walk, a limited range of movement, deformed calf muscles, and breathing problems. Some forms of muscular dystrophy shorten lifespan due to weakness of the breathing muscles and/or heart. To learn more about muscular dystrophy, click here.

A common treatment for muscular dystrophy is the use of steroids, which are able to slow down progression of the disease and can help patients maintain muscle tone. However, steroids eventually lose their effectiveness over time as disease progresses, and raloxifene and tamoxifen have some advantages when compared to them. Both of these new medications have a distinct mechanism of action compared to steroids; they are selective estrogen receptor modulators (SERMs). While steroids are valuable because they help maintain muscle structure, they are less able to maintain muscular function. SERMs are able to maintain function at a far better rate than steroids and also have the added value of improving bone density. A major side effect of steroids is their ability to exacerbate bone issues like osteoporosis and can increase the risk of breaking a bone.

Tamoxifen and raloxifene were also valuable because they helped improve function in the heart and respiratory system; when these muscle systems begin to weaken, it can often mean death for a person with muscular dystrophy. Therefore, maintenance of these muscles is essential to prolong survival. The next step is to begin trials for these medications on human patients. Doctors warn that there could be differences in how men and women may respond since the drugs modulator estrogen, but the benefits will hopefully outweigh these concerns.

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