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Gene Transfer Clinical Trial for Spinal Muscular Atrophy Type 1 This study is a phase I, single-site, dose escalation study to evaluate the safety and efficacy of gene transfer for Spinal Muscular Atrophy Type 1 (SMA1).
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Document Date: 2014-05-01 04:18:01
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Gene Transfer Clinical Trial for Spinal Muscular Atrophy Type 1 This study is a phase I, single-site, dose escalation study to evaluate the safety and efficacy of gene transfer for Spinal Muscular Atrophy Type 1 (SMA1).