The Phase 1
study of ISIS - SMNRx is a
single - dose, dose - escalation
study designed to assess the safety, tolerability and
pharmacokinetic profile of the drug in children with SMA between the ages of 2 - 14 who are medically stable.
Due to the sex - related disparities in
pharmacokinetics and bioavailability results reported by different authors (Soldin, Chung, & Mattison, 2011; Soldin & Mattison, 2009), the choice of a
single genus was chosen to avoid a high dispersion in the data, and the availability of healthy young adult female volunteers for the
study.