Presentation Information
[O24-01]Real-World Assessment of Onasemnogene Abeparvovec in Patients with SMA: Interim report of RESTORE in Japan
○Kayoko Saito1, Misako Sano2, Kamal Benguerba3, Ken Tsuchida2, Rie Teshima2, Sandra P. Reyna4, Omar Dabbous4, Richard S. Finkel5 (1.Institute of Medical Genetics, Tokyo Women's Medical University, Tokyo, Japan, 2.Novartis Pharma K.K., Tokyo, Japan, 3.Novartis Gene Therapies Switzerland GmbH, Rotkreuz, Switzerland, 4.Novartis Gene Therapies, Inc., Bannockburn, IL, USA;, 5.Center for Experimental Neurotherapeutics, St.Jude Childrens Research Hospital, Memphis, TN, USA)