Interim report on the safety and efficacy of longer-term treatment with nusinersen in later-onset spinal muscular atrophy (SMA): Results from the shine study by Kirschner, J, Darras, B.T, Farrar, M.A, Mercuri, E, Chiriboga, C.A, Kuntz, N.L, Shieh, P.B, Tulinius, M, Montes, J, Reyna, S.P, Gambino, G, Foster, R, Bhan, I, Wong, J, Farwell, W

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Nusinersen in infants who initiate treatment in a presymptomatic stage of spinal muscular atrophy (SMA): Interim results from the phase 2 nurture study by Ryan, M.M, De Vivo, D.C, Bertini, E, Hwu, W.L, Crawford, T.O, Swoboda, K.J, Finkel, R.S, Kirschner, J, Kuntz, N.L, Parsons, J, Butterfield, R.J, Topaloglu, H, Omran, T. Ben, Sansone, V.A, Jong, Y.J, Shu, F, Foster, R, Bhan, I, Fradette, S, Farwell, W

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Onasemnogene abeparvovec gene-replacement therapy (GRT) for spinal muscular atrophy type 1 (SMA1): Global pivotal phase 3 study program (STR1VE-US, STR1VE-EU, STR1VE-AP) by Mercuri, E, Baranello, G, Day, J.W, Bruno, C, Corti, S, Chiriboga, C.A, Crawford, T.O, Darras, B.T, Finkel, R.S, Connolly, A.M, Iannaccone, S.T, Kuntz, N.L, Masson, R, Peña, L.D.M, Baldinetti, F, Schultz, M, Shieh, P.B, Smith, E.C, Saito, K, Scoto, M, Spector, S.A, Truncated, A. Authors, Sproule, D.M, Mendell, J.R, Muntoni, F

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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) by 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, Kernbauer, E, Whittle, J, L'Italien, J, Kaspar, B, Sproule, D.M, Spector, S.A, Feltner, D.E, Mendell, J.R

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A classical-path surface-hopping study of Mu, H and T hot-atom collisions with F2 by Jakubetz, Werner, Connor, J N. L., Kuntz, Philip J.

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