First ever therapy for deadly type of muscular dystrophy

CORAL GABLES, Fla. – There’s new hope for children born with a fatal form of muscular dystrophy.

Nicklaus Children’s Hospital is one of the first in the nation and one of only three hospitals in Florida offering Elevidys, an FDA approved infusion medication for the treatment of Duchenne Muscular Dystrophy.

“So the goal is that you stop the disease progression so that the patients can maintain the muscle function they have up and until that point so obviously the younger the patient and the stronger they still are the better the outcome is going to be but the goal is to stop the disease progression and hopefully make them even a little bit stronger and have that last for as long as possible,” said Dr. Migvis Monduy, Medical director of the Neuromuscular and Movement Disorders Programs at Nicklaus.

She said the treatment is currently approved for pediatric patients ages 4 through five with Duchenne Muscular Dystrophy.

Research is ongoing for use in older pediatric patients.

About the Authors:

Kristi Krueger has built a solid reputation as an award-winning medical reporter and effervescent anchor. She joined Local 10 in August 1993. After many years co-anchoring the 6 p.m. and 11 p.m., Kristi now co-anchors the noon newscasts, giving her more time in the evening with her family.

Veteran journalist Kathleen Corso is the special projects producer for Local 10 News.