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Intrathecal administration of AVXS-101 gene-replacement therapy (GRT) for spinal muscular atrophy type 2 (SMA2): Phase 1/2A study (strong)
Finkel, R.S., Day, J.W., Darras, B.T., Kuntz, N.L., Connolly, A.M., Crawford, T.O., Butterfield, R.J., Shieh, P.B., Tennekoon, G., Iannaccone, S.T., Meriggioli, M., Ogrinc, F.G., Kavanagh, S., KernbauVolume:
405
Journal:
Journal of the Neurological Sciences
DOI:
10.1016/j.jns.2019.10.322
Date:
October, 2019
File:
PDF, 200 KB
2019