UF doctors cast net to help muscular dystrophy patients

Patients and families affected by muscular dystrophy urgently need to take practical steps to help slow the destructive course of the disease, say University of Florida researchers who today (Sept. 2, 2005) have launched a Web-based effort to collect information from people coping with the illness.

"The reason that it is so important to identify things that may offer a margin of benefit is that the realistic time horizon for a real treatment or cure is within decades, maybe sooner," said Dietrich Gravenstein, M.D., an anesthesiologist associated with UF's College of Medicine. "I have experience with individuals that suffer from rare diseases. I see how stoic the parents and patients are that face these challenges and I have been frustrated by how little scientifically validated advice there is to simply help them confidently make the many choices they face."

Muscular dystrophies, genetic diseases characterized by progressive weakness of the muscles that control movement, affect between 50,000 to 250,000 people at any one time, according to the Muscular Dystrophy Family Foundation. In Duchenne muscular dystrophy, which affects 1 out of every 3,500 to 5,000 boys, the disease starts to tear down muscles at birth and is typically diagnosed before age 6. Most patients are wheelchair-dependent by 12. By their late teens, most require a respirator to breathe. Victims of the disease do not live past their 20s.

The Web site - www.UFanswers.org - will collect voluntary submissions about personal experiences, lifestyles and the progression of the disease. Even families who have lost loved ones can contribute their experiences and help others living with muscular dystrophy. The Web site officially launches today, just before the annual Jerry Lewis Labor Day Telethon for Muscular Dystrophy begins on Sunday.