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Updated 09/11/2010 05:00 AM

Healthy Living: Study Offers New Hope for Muscular Dystrophy Patients

By: Casey J. Bortnick

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It is an aggressive, debilitating muscle disease for which there is no cure. It attacks young boys when they are toddlers and can put them in a wheelchair by the time they are teenagers.

Even with today’s best treatments, Duchenne muscular dystrophy is often fatal when a patient reaches his 20s or early 30s.

A five-year study at the University of Rochester could help those with this genetic defect live longer.

At first glance, Brayden Juzwiak looks like any regular 6-year-old. He has a lot of energy and never misses a chance to have fun because, for Brayden, time really is precious.

What his parents thought was just slow development was eventually diagnosed as Duchenne muscular dystrophy. The genetic disorder hits boys as toddlers, and attacks the leg muscles and respiratory system. The disease can also lead to heart failure.

"Some people would say it's the worst disease of mankind; it's certainly the most severe," said Dr. Robert Griggs, University of Rochester Medical Center.

Twenty years ago, Dr. Griggs and his team at the University of Rochester discovered that a steroid called Prednisone could slow the progression of the disease.

"It is of dramatic benefit. Patients used to die in their teens and are now living into their 30s," Dr. Griggs said.

The steroid has helped patients like Brayden maintain strength and improve motor function, but it does have side effects like weight gain and difficulty sleeping.

Dr. Griggs said a new steroid called Deflazacort, currently being used in Europe, may do just that, and his team recently received an $11 million grant to prove it.

Griggs will lead a five-year study that will compare three treatment regiments to determine which is best.

"If the Deflazacort turns out to be preferable, we're going to be working to make sure it becomes available in this country for our patients," the doctor explained.

It's welcoming news for a family who knows how important each day really is.

"We just want to make sure that he lives a great life and just does normal things," said Todd Juzwiak, Brayden's father.

Dr. Griggs hopes to begin enrolling 300 patients in the study by next summer. He said only boys who have not yet started a treatment regimen are eligible.