SEC Filings

AVEXIS, INC. filed this Form 8-K on 12/13/2017
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Phase 1 Trial: Key Enrollment Criteria 7 Inclusion 60 months of age and younger at day of vector infusion as defined by the following features: Bi-allelic SMN1 gene deletion 3 copies of SMN2 gene Ability to sit unassisted for 10 or more seconds Negative gene testing for SMN2 gene modifier mutation (c.859G>C) Onset of clinical signs and symptoms consistent with SMA at less than 12 months of age Exclusion Historical or current ability to stand or walk Use of invasive ventilatory support Use or requirement of non-invasive ventilatory support for 12 or more hours daily over the two weeks prior to dosing Key Enrollment Criteria Kaufman et al. Prospective cohort study of spinal muscular atrophy types 2 and 3; Neurology. 2012 Oct 30; 79(18): 1889–1897.



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