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In her role as director of the Scientific Center for Rare Disease at Connecticut Childrens Research Institute, Germain-Lee adds, in addition to helping to tackle the global problem of obesity, the potential for seeing infants and children with SMA having their lives transformed by myostatin inhibitors is truly amazing. February 28 marks National Rare Disease Day.
It does not only influence one organ or hormone pathway but affects how every cell in the body produces and uses energy
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