Sep 26, 2022

New study allows scientists to test therapeutics for rare neurodegenerative disease affecting young children

Posted by in categories: biotech/medical, genetics, neuroscience

For the first time, scientists will be able to test therapeutics for a group of rare neurodegenerative diseases that affect infants and young children, thanks to a new research model created by scientists at the University of Wisconsin-Madison. Their results are published in the Proceedings of the National Academy of Sciences.

Hereditary spastic paraplegias (HSPs) are a group of caused by . They lead tens of thousands of children to develop increased muscle tone in their lower extremities, causing weakness in their legs and ultimately affecting their ability to crawl or walk.

“Kids as early as six months of age that have these start to show signs of disease,” says Anjon Audhya, a professor in the Department of Biomolecular Chemistry at UW-Madison. “Between two and five years of age, these kids become wheelchair-bound, and they unfortunately will never be able to walk.”

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