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Published on Apr 28, 2014
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This video describes the daily struggles and frustrations faced by patients with a rare disease called Classic Galactosemia. Despite a lifelong galactoserestricted diet which is the current mainstay of treatment patients remain uncertain about their prognosis because complications such as infertility and uncoordinated movements can occur later in life. With the support from NIH Common Fund researchers at the University of Utah are now collaborating with scientists at the National Center for Advancing Translational Sciences NCATS to develop a safe and improved therapy in the hope that galactosemic patients can live a healthy and symptomfree life in the future.