AVXS-101 Gene-Replacement Therapy in Presymptomatic Spinal Muscular Atrophy (SMA): Study Update

Meredith Schultz

Presentation information

Oral Presentation

Spinal muscular atrophy

[O28] Spinal muscular atrophy 2

Sat. Jun 1, 2019 11:10 AM - 12:10 PM Room 8 (Conference Room 131+132, 3F, Bldg. 1)

Chair: Hiroyuki Awano (Department of Mental Health for Children and Parents, Kobe University Hospital), Akihiko Ishiyama (National Center Hospital of Neurology and Psychiatry)

[O-168] AVXS-101 Gene-Replacement Therapy in Presymptomatic Spinal Muscular Atrophy (SMA): Study Update

Meredith Schultz¹, Kathryn Swoboda², Michelle Farrar³, Hugh McMillan⁴, Julie Parsons⁵, Kayoko Saito⁶, Mari Matsuo⁶, Tamaki Kato⁶, Courtney Wells⁷, Francis G. Ogrinc⁷, Bryan E. McGill⁷, Douglas E. Feltner⁷, James LItalien⁷, Douglas M. Sproule⁷, Kevin A. Strauss⁸ (1.University of Wisconsin School of Medicine and Public Health, 2.Massachusetts General Hospital, 3.Department of Neurology, Sydney Childrens Hospital, 4.Childrens Hospital of Eastern Ontario, 5.Childrens Hospital Colorado, 6.Tokyo Womens Medical University, 7.AveXis Inc., 8.Clinic for Special Children)

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