Providing Integrated Care for Duchenne Muscular Dystrophy
With its unique interdisciplinary and proactive approach, the Neuromuscular Comprehensive Care Center at Cincinnati Children's has become a premier center for the treatment of Duchenne muscular dystrophy. According to its director, Brenda Wong, MD, the center's approach is essential not only to the quality of clinical care but also to the quality of data that can be obtained through research.
Dr. Wong with one of her patients in the Neuromuscular Comprehensive Care Center.
The Neuromuscular Comprehensive Care Center is involved with ongoing neuromuscular research dealing with Duchenne muscular dystrophy and spinal muscular dystrophy. 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 PTC124 as an Oral Treatment for Nonsense-Mutation- Mediated Duchenne Muscular Dystrophy - PTC Therapeutics
- MRI of mdx mice and Duchenne Muscular Dystrophy boys – pilot studies
- Genomics of Muscular Dystrophy (Divisional research)
Linda Cripe, MD, a cardiologist in the Heart Institute at Cincinnati Children's, explains that the care and treatment of children with Duchenne muscular dystrophy was traditionally provided by a neurologist in a clinic away from a pediatric setting. At Cincinnati Children's, the disease is treated by more than 17 specialties that offer holistic, integrated care of the various medical, social and emotional issues associated with neuromuscular patients. The team includes physicians and subspecialists from such areas as cardiology, endocrinology, genetics, neurology, orthopaedics, psychiatry, physical therapy, pulmonary medicine and many others who come together to treat the whole child.
Dr. Wong says the integrated approach to treatment at the center is essential to conduct meaningful quality research. Clinical trials on neuromuscular disease require a minimum standard of care to be effective. When an institution 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 not just in receiving treatment but also want to be involved in clinical trials."
"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 here to lead the world with regard to clinical trials and basic science research to help find a cure for this devastating illness."
