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Kennedy Krieger Institute Opens Center for Genetic Muscle Disorders
Kennedy Krieger Institute announced today the opening of the new Center for Genetic Muscle Disorders that will improve and expand services to individuals with muscle disorders such as muscular dystrophy and congenital myopathy in the Baltimore/Washington region and across the nation. The Center provides expert clinical care to children and adults with these disorders, and offers leading research programs both in the clinical and laboratory setting.
Children with genetic muscle disorders face loss of muscle mass and strength, though the rate of progression and symptoms vary according to the type of disease. Individuals with these disorders face a range of challenges, which may include declining quality of life, shortened life expectancies, and heart and breathing conditions. The collective expertise of the Center’s staff provides these patients with a succinct and informed diagnosis and prognosis, as well as a unique perspective on how the disease will impact their day-to-day life. As leaders in their fields, staff members are able to work with patients to find practical solutions to the specific problems they are experiencing as well as frequently slow the course of the disease.
“Our new Center serves an important need within the muscle disorders community,” said Kathryn R. Wagner, M.D., Ph.D., Director of the Center for Genetic Muscle Disorders. “Unlike some facilities that may be unable to serve the adult population, our Center maintains a continuum of care for patients transitioning from childhood to adulthood and we can address all of the issues they may face.”
Due to the collaboration between Kennedy Krieger’s various programs, patients who visit the Center also have easy access to a variety of clinical services to help manage their symptoms and improve their quality of life, including physical therapy, occupational therapy, aquatic therapy and assistive technology. In addition, the Center is a Muscular Dystrophy Association (MDA) Clinic, giving patients direct access to a wide variety of MDA services such as support groups and equipment loan programs.
To learn more about the Center and how it’s serving patients, listen to Dr. Wagner’s podcast on Kennedy Krieger’s Web site: http://podcasts.kennedykrieger.org/.
A research leader
Another priority for the Center is playing an active role in research. While the genetic basis is known for most genetic muscle disorders, there are no cures and patients have few treatment options. Active participation in research programs will allow the Center to better serve patients by providing direct access to treatment clinical trials for which patients may be eligible. The Center for Genetic Muscle Disorders is already engaged with research in the following areas:
- The Center is working with Facioscapulohumeral muscular dystrophy (FSH) patients and their families to characterize genetic abnormalities and expressions. This will help determine the genetic differences between mildly affected (or unaffected) family members and severely affected family members.
- Clinical research is being conducted on myostatin, a natural inhibitor of muscle growth present in all animals and humans. Blocking myostatin can result in increased muscle growth, increased regeneration of muscle and decreased muscle scarring. The Center is investigating pharmacological agents that are in development as new treatments for muscular dystrophies.
- The Center will soon initiate a clinical trial in a potential treatment for Duchenne Muscular Dystrophy called sildenafil. The drug, more commonly known as Viagara, has shown potential to help treat heart and skeletal muscle damage in animal models.
About Genetic Muscle Disorders
There are more than 30 genetic disorders of muscle, which are all marked by progressive wasting and weakness of muscle tissue. Muscular dystrophy and congenital myopathy are two categories of inherited myopathies. The majority of disorders begin in childhood, though the rate of progression and associated symptoms vary according to the disease. For example, some types of muscular dystrophy are multi-system disorders that affect the heart, gastrointestinal and nervous systems, endocrine glands, skin, eyes and other organs.
About the Kennedy Krieger Institute
Internationally recognized for improving the lives of children and adolescents with disorders and injuries of the brain, spinal cord and muscle, the Kennedy Krieger Institute in Baltimore, MD serves more than 13,000 individuals each year through inpatient and outpatient clinics, home and community services and school-based programs. Kennedy Krieger provides a wide range of services for children with developmental concerns mild to severe, and is home to a team of investigators who are contributing to the understanding of how disorders develop while pioneering new interventions and earlier diagnosis. For more information on Kennedy Krieger Institute, visit www.kennedykrieger.org.