Funding to break the barrier to participation in spinocerebellar ataxia research

Congratulations to Dr. Ian Harding, Department of Neuroscience, on being awarded a $50,000 National Ataxia Foundation Young Investigator Award for research in spinocerebellar ataxias.

Spinocerebellar ataxias (SCA) are progressive, life-shortening inherited neurological disorders. People with ACS experience a wide range of different symptoms, including difficulty coordinating movement, speaking, mental functions like thinking, and managing their emotions. Currently, no treatment exists to stop the progression of the disease.

Dr Harding said: “Our project has three main aims, each addressing a major current limitation in spinocerebellar ataxia (SCA) research.”

First, the study will use simple online assessments of hand movements, speech, thinking skills and mood that can be done on a computer or tablet from home. “By using web-based platforms, we can remove many barriers to research participation because people can undertake the assessments where they feel comfortable.

“This is essential in rare diseases like ACS to ensure adequate sample sizes and the participation of people from all walks of life.

“Second, our project seeks to define and track not only movement deficits in people with ACS, but also cognitive and psychiatric symptoms.”

He said that current clinical management largely focuses on movement deficits in ACS, but growing evidence and patient testimonials indicate that these other disease features also have a significant impact on their quality. of life.

“Finally, we will focus not only on long-term symptom changes that occur over a year or more, but also on short-term variability in symptom severity that occurs on the order of weeks to a few weeks. month.

“To date, research has focused exclusively on the long-term changes that result from neurodegeneration, but understanding the cause and extent of short-term variability is essential for patient care and for considering variability in treatment trial endpoint measures.

“Taken together, we are pioneering a paradigm shift in the way behavioral monitoring is undertaken in ACS, with the potential to provide new symptom monitoring tools for use in clinical and research settings and new insights into symptom expression and variability to inform clinical practice and clinical trials. design.”

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