Newswise BALTIMORE, MD (April 12, 2017) Three grassroots foundations founded by the families of young boys diagnosed with Duchenne muscular dystrophy (DMD) have jointly awarded a $100,000 grant to Kennedy Krieger Institutes Center for Genetic Muscle Disorders for research into novel drug therapies. The funding from Ryans Quest, of New Jersey, along with Michaels Cause and Pietros Fight, both of New York, will enable the scientific team to study existing drugs that have shown early therapeutic promise in cell cultures. "We remain dedicated to helping identify existing FDA-approved drugs that can be repurposed to help treat Duchenne muscular dystrophy and ultimately expedite potential therapies for patients," said David Schultz, co-founder of Ryans Quest and father of 11-year-old Ryan, who has DMD. Researcher Dr. Kathryn Wagner, and her colleagues Gabsang Lee and Congshan Sun, recently screened existing drugs using human myoblasts derived from induced pluripotent stem cells (iPSCs) from individuals living with DMD. The iPSCs are a type of stem cell that can be generated from mature living cells. Several drugs reliably produced a positive therapeutic response in the cells in culture. With this funding, the findings will be validated and trialed in the mouse model for DMD. As many of … Continue reading
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