International Initiative Underway in Rochester to Enhance Muscular Dystrophy Therapies Globally
The University of Rochester Medical Center is spearheading a significant study on Duchenne muscular dystrophy (DMD), a rapidly progressing disease that primarily affects children. Robert "Berch" Griggs, M.D., is leading the research, with the aim of generating valuable insights into the disease's progression and impact on patients.
The study focuses on three commonly used treatments: a daily dose of prednisone, a prednisone regimen with alternating periods without the drug, and deflazacort. A total of 300 boys aged 4 through 7 from North America and Europe will be included in the study.
In the United States, Barbara Herr, assistant professor of Neurology, oversees the project, while 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, will also contribute from Rochester. Michael McDermott, Ph.D., and Rabi Tawil, M.D. are co-leaders of biostatistical analysis and data management for the study.
The study seeks to eliminate the chaos in the current treatment landscape for DMD by providing critical data on how symptoms evolve and affect patients over time. This information will be invaluable for clinical trials and therapeutic development.
Researchers will evaluate children every six months for three to five years, measuring their breathing capacity, satisfaction with treatment, and time to stand up after lying down. The study aims to find the best balance between effective treatment and side effects for daily prednisone, a treatment that boosts muscle strength dramatically in patients with DMD.
It is worth noting that about two-thirds of Duchenne muscular dystrophy cases occur in families with a history of the disease, while one-third occur spontaneously. Side effects of daily prednisone include weight gain, behavioral effects such as hyperactivity or irritability, loss of bone, and potential fractures.
Griggs is at the forefront of the international effort to find an effective treatment for Duchenne muscular dystrophy and has recently been asked to comment on new findings from a study of the drug cyclosporine. His expertise in the field is widely recognised.
Untreated, boys with Duchenne muscular dystrophy end up in a wheelchair before they become teenagers. The study's findings will undoubtedly contribute to improving clinical knowledge of the disease and informing both clinical practice and future research directions in neuromuscular disorders related to DMD.
This study brings together two worldwide groups of physicians who specialize in the study of muscle disorders: The Muscle Study Group, based in Rochester and headed by Griggs, and the TREAT-NMD study group, a consortium of institutions across Europe and elsewhere. The collaboration promises to yield groundbreaking results in the fight against Duchenne muscular dystrophy.
References: 1. Duchenne muscular dystrophy study 2. Understanding the burden of Duchenne muscular dystrophy
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