WMS Poster 2018: SMN protein levels before and after treatment with risdiplam (RG7916) in patients with Type 1 to 3 SMA compared to healthy subjects
SMN protein levels were compared from whole blood in patients across SMA types versus healthy subjects. SMN protein samples were collected from patients participanting across the various risdiplam (RG7916) studies: FIREFISH, SUNFISH, JEWELFISH. SMN protein levels were compared between copy numbers, across a wide age range (3.5 months to 52 years in patients with SMA), and in longitudinal data for patients on treatment with RG7916 versus patients on placebo.
Receive this presentation and more content via email