Healthy Kids: New hope for kids with Duchenne muscular dystrophy after FDA grants gene therapy approval
COLORADO SPRINGS, Colo. (KRDO) -- A groundbreaking gene therapy was recently approved by the U.S. Food and Drug Administration to treat the most common form of muscular dystrophy in children, Duchenne muscular dystrophy, for kids ages four and five.
Doctors say it is the biggest advancement in years.
"The new gene therapy for Duchenne muscular dystrophy is a major breakthrough," said Dr. Jerry Mendell, with Nationwide Children's Hospital. "It is the first time we have, I would say, a treatment that addressed the fundamental missing protein for the disease."
The first clinical trials for this treatment conducted through Nationwide Children's Hospital demonstrated life-changing impacts for kids with this devastating, progressive disease that affects mobility, heart, and respiratory function.
Duchenne muscular dystrophy, also known as DMD, typically affects boys and begins in utero. As the disease progresses muscle weakness and atrophy spread across the body and most individuals require a wheelchair by their teenage or early adult years. It usually presents itself when boys are toddlers.
"The disease becomes obvious because the boys have trouble meeting developmental motor milestones, says Dr. Mendell. "They sit late, they stand late, they walk late and that is the first indication to the parent that they need to be seen by their doctor."
Dr. Mendell has dedicated his 50-year career to understanding and treating DMD, the most common form of muscular dystrophy. It's a genetic disorder characterized by progressive muscle degeneration and weakness due to the alterations of a protein that helps keep muscle cells intact. Dr. Mendell and his team received FDA approval this summer for developed gene therapy to correct the cell function of those with the disease.
"Sometimes it is humbling to see how grateful they are in terms of what we can do now," said Dr. Mendell. "It is imperfect, it will be better as time goes on, there are ways of improving on gene therapy and we will see that. This is really pioneering work and really the foundation for gene therapy and this disease but there will be improvements."
This treatment brings hope for a brighter future for children born with this genetic disease. Another step would be expanding the treatment for kids of all ages. Right now the program only has FDA approval for children ages four and five.
