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THURSDAY, Nov. 15 (HealthDay News) -- When injected into mice, a novel compound eliminated the severe muscle stiffness and weakness that is a disabling symptom of one form of muscular dystrophy.
But the research, though promising, is still in its infancy, the investigators and other experts stressed.
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"It's an important step forward that should be encouraging for people with the condition and their families and it shows that doctors and scientists are really now beginning to fix their attention on fixing this disease, rather than trying to understand what goes wrong," said study senior author Dr. Charles Thornton, a professor of neurology at the University of Rochester School of Medicine and Dentistry.
Thornton cautioned that he would not use the term "breakthrough" to describe the results, however.
"It's an important first step and these results are encouraging that effective therapies for myotonic [muscle-stiffening] dystrophy can be developed that can actually ameliorate some of the clinical symptoms," added Dr. Valerie Cwik, medical director and vice president of research at the Muscular Dystrophy Association (MDA). "But this is a study in mice, and we know from lots of other disorders and studies in mice that what works in mice doesn't always translate into effective therapy in humans."
According to the MDA, myotonic dystrophy is one of nine forms of muscular dystrophy, a group of genetic, degenerative and potentially disabling diseases which primarily affect voluntary muscles.
Myotonic dystrophy type 1 is the most common form of muscular dystrophy affecting adults. It is caused when a section of DNA is repeated on either chromosome 19 or chromosome 3.
"Myotonic dystrophy affects multiple systems in the body, so it causes central nervous system problems with altered cognition in some people, sleep disturbances, cataracts, droopy eyelids, not only myotonia," Cwik explained. Myotonia involves a "difficulty relaxing muscles but also muscle weakness, which is actually more disabling for many people," she said. It also involves "intestinal problems and, importantly, it can affect the heart, with potentially life-threatening arrhythmias," Cwik explained. "It's a multi-system disorder."
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