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Better CMT outcome measurement is Wayne State University physician's goal
Date:12/7/2011

ssion and anger.

"We want something that enables children to tell us how this disease impacts their lives at any given point," she said. Questionnaires will be given to all children who come to Wayne State's CMT clinic through the consortium. Scores will be aligned with physical functioning and electrophysiologic studies to spot correlations between how patients feel they are doing and how the disease actually is impacting them neurophysiologically.

"We have shown that quality-of-life scores are low in children with CMT compared to healthy children," Ramchandren said. "We are hoping to show that if those scores change dramatically over the next two to three years, then quality of life might be an outcome measure that can be used in a clinical trial."

Such methodology has worked very well in cancer research, she said, where sometimes quality-of-life scores are first to improve, and then over time researchers see changes in tumor size or disease physiology. Ramchandren believes the same approach could be used for CMT and is working with mentor Lawrence Lum, M.D., professor of oncology at WSU and the Barbara Ann Karmanos Cancer Institute, on early phase clinical trial methodology training. Researchers there take bench work from animal models and translate it into new cancer therapies, she said.

Her eventual goal is to develop a clinical trials unit for all neuromuscular disorders, capable of translating promising animal research into new treatments.


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Contact: Julie O'Connor
julie.oconnor@wayne.edu
313-577-8845
Wayne State University - Office of the Vice President for Research
Source:Eurekalert

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