(Q63319636)
Statements
SMNRx (English)
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An Open-label, Escalating Dose Study to Assess the Safety, Tolerability and Dose-range Finding of a Single Intrathecal Dose of ISIS 396443 in Patients With Spinal Muscular Atrophy (English)
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30 November 2011
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31 January 2013
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28
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2 year
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14 year
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