Clinical Edge

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AVXS-101 May Result in Long-Term Motor Improvements in SMA

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.