New Frontiers in the Treatment of Spinal Muscular Atrophy
- PMID: 30376223
New Frontiers in the Treatment of Spinal Muscular Atrophy
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Single-Dose Gene-Replacement Therapy for Spinal Muscular Atrophy.N Engl J Med. 2017 Nov 2;377(18):1713-1722. doi: 10.1056/NEJMoa1706198. N Engl J Med. 2017. PMID: 29091557 Clinical Trial.
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Nusinersen versus Sham Control in Infantile-Onset Spinal Muscular Atrophy.N Engl J Med. 2017 Nov 2;377(18):1723-1732. doi: 10.1056/NEJMoa1702752. N Engl J Med. 2017. PMID: 29091570 Clinical Trial.
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