Most people with Fanconi anemia are diagnosed before they turn 12 but don’t live past 30. Wei Du—a researcher in the WVU School of Pharmacy and the WVU Cancer Institute—is exploring the metabolic processes the underlie this form of anemia. Her findings may lead to new gene therapies that help patients live better, longer. Treating […]
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Tailor-made drug appears to REVERSE symptoms in girl with rare disease
World first as experimental drug appears to REVERSE symptoms in girl, 7, with incurable disease that causes paralysis, blindness, dementia and early death Mila Makovec, from Boulder, Colorado, was born happy and healthy, but soon she developed a stutter, had trouble walking, and became blind At age six, Mila was diagnosed with Batten disease – […]