Sma – therapy

Spinal muscular atrophy (SMA) is caused by deletion/mutation of the survival motor neuron 1 gene (SMN1). AVXS-101 IT is a one-time gene therapy designed to deliver fully functional copy of human SMN gene to address the genetic root cause of SMA. STRONG (CL-102, NCT03381729), a phase 1, open-label study, assesses the safety, optimal dose, and efficacy of AVXS-101 IT. Patients (pts; biallelic SMN1 loss, 3xSMN2) aged ≥6

Source: Neuromuscular Disorders - September 28, 2020 Category: Neurology Authors: D. Chand, R. Finkel, J. Day, B. Darris, N. Kuntz, A. Connolly, C. Zaidman, T. Crawford, R. Butterfield, P. Shieh, G. Tennekoon, J. Brandesma, S. Iannaccone, M. Meriggioli, S. Tauscher-Wisniewski, J. Shoffner, F. Ogrinc, S. Kavanagh, D. Feltner, J. Mendell Source Type: research