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A drug called nusinersen (Spinraza) is now in the hands of doctors across the nation, who will use it, most urgently, to treat young children with a severe and potentially fatal illness called spinal muscular atrophy (SMA). The drug was conceived and tested over several years in mouse models of SMA by Professor Adrian Krainer, Ph.D., and his colleagues at Cold Spring Harbor Laboratory (CSHL) in collaboration with Dr. Frank Bennett and his team at Ionis Pharmaceuticals. News of the approval pleased the drug's developers and delighted parents of affected children. Read more
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