Long-term durability of Zolgensma® in patients with SMA Typ 1

Avexis and Novartis present new data at AAN 2019.

As of March 8, 2019, all patients maintained the motor function and milestones gained during the trial following treatment with Zolgensma. In addition, no patients had any additional requirements for ventilatory or nutritional support.

Two of the four patients who required Bilevel Positive Airway Pressure (BiPAP) support at the beginning of the long-term follow-up study no longer required it regularly. Read the full study data