Interim Efficacy and Safety Results from the Phase 2 NURTURE Study Evaluating Nusinersen in Presymptomatic Infants with Spinal Muscular Atrophy (S46.003)

Conclusions:The NURTURE interim analysis shows that all the infants treated with nusinersen are alive and generally achieving new motor skills and appropriate age-related developmental gains. The efficacy and safety profile of nusinersen supports continued clinical development.Study Supported by: BiogenDisclosure: Dr. De Vivo has received personal compensation for activities with AveXis, Biogen, Roche, IONIS, Sarepta, Cytokinetics Pharmaceuticals, and the SMA Foundation. Dr. De Vivo has received research support from NIH, DOD, SMA Foundation,and Hope for Children Research Foundation. Dr. Hwu has nothing to disclose. Dr. Reyna has received personal compensation for activities with Biogen as an employee. Dr. Reyna holds stock and/or stock options in Biogen. Dr. Farwell has received personal compensation for activities with Biogen Idec as an employee. Dr. Farwell holds stock and/or stock options in Biogen Idec. Dr. Gheuens has received personal compensation for activities with Biogen as an employee. Dr. Sun has received personal compensation for activities with Biogen as an employee. Dr. Sun hold stock and/or stock options in Biogen. Dr. Zhong has received personal compensation for activities with Biogen Idec. Dr. Zhong holds stock and/or stock options in Biogen Idec. Dr. Su has received personal compensation for activities with IONIS Pharmaceuticals Inc. as an employee. Dr. Su holds stock and/or stock options in IONIS Pharmaceuticals. Dr. Schneider has received personal compens...

Source: Neurology - April 17, 2017 Category: Neurology Authors: De Vivo, D. C., Hwu, W.-L., Reyna, S. P., Farwell, W., Gheuens, S., Sun, P., Zhong, Z. J., Su, J., Schneider, E., Bertini, E., on behalf of the NURTURE Study Investigators Tags: Child Neurology: Molecular Biology to Clinical Trials Source Type: research