Unique Approach to Treating Duchenne Muscular Dystrophy
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Duchenne muscular dystrophy is the most common form of muscular dystrophy. One in 3,000 boys has this fatal, genetic disease. It is typically diagnosed in children ages 3 to 7. |
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Linda Cripe, MD, a cardiologist in The Heart Center at Cincinnati Children's, explains that the care and treatment of Duchenne muscular dystrophy was traditionally provided by a neurologist in a clinic away from a children's hospital.
At Cincinnati Children's, the disease is treated by more than 17 specialists who offer integrated care of the various medical, social and emotional issues associated with neuromuscular patients.
The team includes physicians and subspecialists from areas such as cardiology, endocrinology, genetics, neurology, orthopaedics, psychiatry, physical therapy, pulmonary medicine and many others who come together to treat the whole child.
With its unique interdisciplinary and proactive approach, the Neuromuscular Comprehensive Care Center at Cincinnati Children's has established a world-class program – a one-of-a-kind program – that is changing the outcome for kids with Duchenne muscular dystrophy. Patients are coming to Cincinnati from all over the world – California, Massachusetts, Mississippi, Florida, Romania and Venezuela, just to name a few.
"Everyone is genuinely kind," says Angela Malone who travels from Mississippi for treatment for her 5-year-old son who has Duchenne muscular dystrophy. "You can tell they really care, and they gave me hope, not false hope, but hope. We have not found that elsewhere. That's why we come so far."
Research and Clinical Care Go Hand in Hand
Brenda Wong, MD, child neurologist and director of the Neuromuscular Comprehensive Care Center, says the integrated approach to treatment is essential to conduct meaningful, quality research. Clinical trials on neuromuscular disease require a minimum standard of care to be effective.
When a medical center such as Cincinnati Children's provides the best and most comprehensive care for its patients, a change during a study can more easily be attributed to the study interventions and not to confounding factors that may arise from lack of medical interventions. "Research and clinical care go hand in hand," she says. "Families are interested in receiving treatment, and they also want to be involved in clinical trials."
The Neuromuscular Comprehensive Care Center is involved with ongoing neuromuscular research dealing with Duchenne muscular dystrophy and spinal muscular atrophy. Current studies include:
- NIH-funded multi-centered American Spinal Muscular Atrophy Randomized Trials (AmSMART)
- NIH-funded multi-centered United Dystrophinopathy Project
- A Phase 2 Study of the drug PTC124 as an Oral Treatment for Nonsense-Mutation-Mediated Duchenne Muscular Dystrophy – PTC Therapeutics
- MRI of mdx mice and boys with DMD – pilot studies
- Genomics of Muscular Dystrophy (Divisional research)
"I think Cincinnati Children's has the potential to make an incredible impact in the care and treatment of children with Duchenne muscular dystrophy," Dr. Cripe says. "We have the ability to lead the world with regard to clinical trials and basic science research to try and find a cure for this devastating illness. And by intervening at an earlier age, at diagnosis, we hope to slow the progression of heart disease and change the natural history of the disease process."
