International initiative spearheaded by Rochester aims to enhance treatments for Muscular Dystrophy

International initiative spearheaded by Rochester aims to enhance treatments for Muscular Dystrophy

The University of Rochester Medical Center has embarked on an international study to improve treatment for Duchenne muscular dystrophy (DMD), a rapidly progressing disease that primarily affects children. Led by Robert "Berch" Griggs, M.D., the study seeks to better understand the disease's progression and the impacts of symptoms on patients and caregivers.

The research, funded by the National Institute of Neurological Disorders and Stroke with at least $11 million, will include 300 boys ages 4 through 7 from North America and Europe. The study's primary goal is to enhance clinical trial design and therapeutic strategies for DMD by gathering comprehensive data on symptom impact from both patients and caregivers.

The study will evaluate children every six months for three to five years, using various measures such as the breathing capacity of patients, satisfaction with treatment, and the time it takes for patients to stand up after lying down. Researchers will also focus on the balance between effective treatment and side effects, particularly those associated with daily prednisone, such as weight gain, behavioral effects, loss of bone, and possible fractures.

Robert "Berch" Griggs is at the forefront of an international effort to find an effective treatment for Duchenne muscular dystrophy. He is joined by co-leaders Michael McDermott, Ph.D., professor of Biostatistics and Neurology, and Rabi Tawil, M.D., professor of Neurology, who will oversee biostatistical analysis and data management for the study. Barbara Herr, assistant professor of Neurology, manages the U.S. portion of the project, while Emma Ciafaloni, M.D., leads the study of patients in Rochester.

The study brings together two worldwide groups of physicians who specialize in the study of muscle disorders: The Muscle Study Group and TREAT-NMD study group. Shree Pandya, lead U.S. physical therapist, Mary Brown, Heart Research Program manager, William Martens, data manager, and Emma Ciafaloni, M.D., associate professor of Neurology, are part of the team from Rochester.

The new study will investigate the three treatments most commonly used today for Duchenne muscular dystrophy. Daily prednisone, one of these treatments, boosts muscle strength by 20 percent and slows the degeneration of muscles in patients with the disease. However, it's crucial to understand the side effects associated with this treatment to ensure optimal care for patients.

Recruiting for the study will begin in the summer of 2011, with the funding expected to reach close to $15 million. The study aims to improve the care of muscular dystrophy patients by providing valuable insights into the disease's progression and the impacts of symptoms, ultimately leading to more effective treatment evaluation and development.

1. The University of Rochester Medical Center is investigating Duchenne muscular dystrophy (DMD) through an international study to improve treatment
2. The study is led by Robert "Berch" Griggs, M.D., with the goal of enhancing clinical trial design and therapeutic strategies for DMD
3. The research will gather data from 300 boys ages 4 through 7 from North America and Europe, funded by the National Institute of Neurological Disorders and Stroke
4. The study's primary goal is to understand the disease's progression and impacts on patients and caregivers
5. The research will evaluate children every six months for three to five years, using various measures like breathing capacity, treatment satisfaction, and time to stand up
6. Researchers will balance effective treatment with side effects, particularly those linked to daily prednisone
7. Side effects include weight gain, behavioral effects, loss of bone, and possible fractures from daily prednisone
8. Michael McDermott, Ph.D., and Rabi Tawil, M.D. will oversee biostatistical analysis and data management for the study
9. Barbara Herr, assistant professor of Neurology, manages the U.S. portion of the research, while Emma Ciafaloni, M.D., leads the study in Rochester
10. Two worldwide groups of physicians specializing in muscle disorders are collaborating: The Muscle Study Group and TREAT-NMD study group
11. Shree Pandya, Mary Brown, William Martens, and Emma Ciafaloni, M.D., are part of the team from Rochester
12. The study will investigate the three treatments most commonly used today for Duchenne muscular dystrophy
13. Daily prednisone boosts muscle strength by 20 percent and slows muscle degeneration in patients with the disease
14. It's crucial to understand the side effects associated with this treatment for optimal care for patients
15. Recruiting for the study begins in the summer of 2011, with funding expected to reach close to $15 million
16. The study aims to provide valuable insights into the disease's progression and impacts, leading to more effective treatment evaluation and development
17. Improved care of muscular dystrophy patients is the ultimate goal of the study
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