SEC Filings

8-K
AVEXIS, INC. filed this Form 8-K on 12/13/2017
Entire Document
 

SMA Types: A Devastating Disease 3 TYPE 1 TYPE 2 TYPE 3 TYPE 4 SMN2 Copy Number Two Three** Three or Four Four to Eight Onset Before 6 Months 6-18 Months Early childhood to early adulthood (juvenile) Adulthood (20s-30s) usually after 30 Incidence per Live Birth Approximately 60% Approximately 27% Approximately 13% Uncommon; limited information available Developmental Milestones Will never be able to sit without support Difficulty breathing & swallowing Can’t crawl/will never walk Will never be able to walk without support Most will never stand without support Stand alone and walk independently, but may lose ability to walk over time Stand alone and walk but may lose ability to walk in 30s-40s Survival <10% Event free* by two years of age 68% alive at age 25 Normal Normal *Event = Death or >16-hr/day ventilation continuously for > 2 wks, in the absence of an acute reversible illness **100% have 3 copies (PNCR)

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