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Single-Dose Gene-Replacement Therapy for Spinal Muscular Atrophy

The New England journal of medicine, 2017-11, Vol.377 (18), p.1713-1722 [Peer Reviewed Journal]

ISSN: 0028-4793 ;EISSN: 1533-4406 ;DOI: 10.1056/NEJMoa1706198

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