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Spinal zap: Isis’ atrophy drug shines in top-line results for kids and babies

By Randy Osborne
Staff Writer

Monday, February 24, 2014
Calling the top-line phase Ia/IIb data with the company’s antisense drug for spinal muscular atrophy (SMA) in children “achingly important” and the benefit in a subset “shocking,” Isis Pharmaceuticals Inc. CEO Stanley Crooke said intrathecal ISIS-SMNRx has proven safe so far, too.

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