Key clinical point: At 2 years, patients with SMA1 who had received a therapeutic dose of AVXS-101 had motor improvement.
Major finding: Seven of 12 treated patients did not need daily noninvasive ventilation at 2 years.
Study details: A phase 1/2a study of 12 patients with SMA1.
Disclosures: Dr. Dabbous is an employee of AveXis, which developed AVXS-101.
Dabbous O et al. CNS 2019. Abstract 199.