Efficacy and safety of nusinersen in infants with spinal muscular atrophy (SMA): Final results from the phase 3 ENDEAR study
Finkel, R, Kuntz, N, Mercuri, E, Chiriboga, C.A, Darras, B, Topaloglu, H, Montes, J, Su, J, Zhong, Z.J, Gheuens, S, Bennett, C.F, Schneider, E, Farwell, W
Published in European journal of paediatric neurology (01.06.2017)
Published in European journal of paediatric neurology (01.06.2017)
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Interim analysis of the phase 3 CHERISH study evaluating nusinersen in patients with later-onset spinal muscular atrophy (SMA): Primary and descriptive secondary endpoints
Mercuri, E, Finkel, R, Kirschner, J, Chiriboga, C.A, Kuntz, N, Darras, B, Shieh, P.B, Saito, K, De Vivo, D.C, Mazzone, E.S, Montes, J, Yang, Q, Zhong, Z.J, Gheuens, S, Bennett, C.F, Schneider, E, Farwell, W
Published in European journal of paediatric neurology (01.06.2017)
Published in European journal of paediatric neurology (01.06.2017)
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Journal Article
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
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.
Published in Journal of the neurological sciences (15.10.2019)
Published in Journal of the neurological sciences (15.10.2019)
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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)
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.
Published in Journal of the neurological sciences (15.10.2019)
Published in Journal of the neurological sciences (15.10.2019)
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Onasemnogene abeparvovec gene-replacement therapy (GRT) in presymptomatic spinal muscular atrophy (SMA): SPR1NT study update
Strauss, K.A., Swoboda, K.J., Farrar, M.A., McMillan, H.J., Parsons, J., Krueger, J.M., Iannaccone, S.T., Chiriboga, C.A., Kwon, J.M., Saito, K., Scoto, M., Baldinetti, F., Schultz, M., Kernbauer, E., Farrow, M., Ogrinc, F.G., Kavanagh, S., Feltner, D.E., McGill, B.E., Spector, S.A., L’Italien, J., Sproule, D.M., Muntoni, F.
Published in Journal of the neurological sciences (15.10.2019)
Published in Journal of the neurological sciences (15.10.2019)
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