Safety and efficacy of olesoxime in patients with type 2 or non-ambulatory type 3 spinal muscular atrophy: a randomised, double-blind, placebo-controlled phase 2 trial

E. Bertini; E. Dessaud; E. Mercuri; F. Muntoni; J. Kirschner; C. Reid; A. Lusakowska; G.P. Comi; J.-M. Cuisset; J.-L. Abitbol; B. Scherrer; P.S. Ducray; J. Buchbjerg; E. Vianna; W.L. van der Pol; C. Vuillerot; T. Blaettler; P. Fontoura; C. André; C. Bruno; B. Chabrol; N. Deconinck; B. Estournet; S. Fontaine-Carbonnel; N. Goemans; K. Gorni; A. Govoni; M. Guglieri; H. Lochmuller; F. Magri; M. Mayer; W. Müller-Felber; F. Rivier; H. Roper; U. Schara; M. Scoto; L. van den Berg; G. Vita; M.C. Walter

doi:10.1016/S1474-4422(17)30085-6

Safety and efficacy of olesoxime in patients with type 2 or non-ambulatory type 3 spinal muscular atrophy: a randomised, double-blind, placebo-controlled phase 2 trial

E. Bertini, E. Dessaud, E. Mercuri, F. Muntoni, J. Kirschner, C. Reid, A. Lusakowska, G.P. Comi, J.-M. Cuisset, J.-L. Abitbol, B. Scherrer, P.S. Ducray, J. Buchbjerg, E. Vianna, W.L. van der Pol, C. Vuillerot, T. Blaettler, P. Fontoura, C. André, C. BrunoB. Chabrol, N. Deconinck, B. Estournet, S. Fontaine-Carbonnel, N. Goemans, K. Gorni, A. Govoni, M. Guglieri, H. Lochmuller, F. Magri, M. Mayer, W. Müller-Felber, F. Rivier, H. Roper, U. Schara, M. Scoto, L. van den Berg, G. Vita, M.C. Walter

Istituto "Giannina Gaslini"

Research output: Contribution to journal › Article › peer-review

Original language	English
Pages (from-to)	513-522
Number of pages	10
Journal	The Lancet Neurology
Volume	16
Issue number	7
DOIs	https://doi.org/10.1016/S1474-4422(17)30085-6
Publication status	Published - 2017

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10.1016/S1474-4422(17)30085-6

https://www.scopus.com/inward/record.uri?eid=2-s2.0-85018331440&doi=10.1016%2fS1474-4422%2817%2930085-6&partnerID=40&md5=e16396ac5be1f7a0a271be70760922bd

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Bertini, E., Dessaud, E., Mercuri, E., Muntoni, F., Kirschner, J., Reid, C., Lusakowska, A., Comi, G. P., Cuisset, J-M., Abitbol, J-L., Scherrer, B., Ducray, P. S., Buchbjerg, J., Vianna, E., van der Pol, W. L., Vuillerot, C., Blaettler, T., Fontoura, P., André, C., ... Walter, M. C. (2017). Safety and efficacy of olesoxime in patients with type 2 or non-ambulatory type 3 spinal muscular atrophy: a randomised, double-blind, placebo-controlled phase 2 trial. The Lancet Neurology, 16(7), 513-522. https://doi.org/10.1016/S1474-4422(17)30085-6

Safety and efficacy of olesoxime in patients with type 2 or non-ambulatory type 3 spinal muscular atrophy: a randomised, double-blind, placebo-controlled phase 2 trial. / Bertini, E.; Dessaud, E.; Mercuri, E.; Muntoni, F.; Kirschner, J.; Reid, C.; Lusakowska, A.; Comi, G.P.; Cuisset, J.-M.; Abitbol, J.-L.; Scherrer, B.; Ducray, P.S.; Buchbjerg, J.; Vianna, E.; van der Pol, W.L.; Vuillerot, C.; Blaettler, T.; Fontoura, P.; André, C.; Bruno, C.; Chabrol, B.; Deconinck, N.; Estournet, B.; Fontaine-Carbonnel, S.; Goemans, N.; Gorni, K.; Govoni, A.; Guglieri, M.; Lochmuller, H.; Magri, F.; Mayer, M.; Müller-Felber, W.; Rivier, F.; Roper, H.; Schara, U.; Scoto, M.; van den Berg, L.; Vita, G.; Walter, M.C.

In: The Lancet Neurology, Vol. 16, No. 7, 2017, p. 513-522.

Research output: Contribution to journal › Article › peer-review

Bertini, E, Dessaud, E, Mercuri, E, Muntoni, F, Kirschner, J, Reid, C, Lusakowska, A, Comi, GP, Cuisset, J-M, Abitbol, J-L, Scherrer, B, Ducray, PS, Buchbjerg, J, Vianna, E, van der Pol, WL, Vuillerot, C, Blaettler, T, Fontoura, P, André, C, Bruno, C, Chabrol, B, Deconinck, N, Estournet, B, Fontaine-Carbonnel, S, Goemans, N, Gorni, K, Govoni, A, Guglieri, M, Lochmuller, H, Magri, F, Mayer, M, Müller-Felber, W, Rivier, F, Roper, H, Schara, U, Scoto, M, van den Berg, L, Vita, G & Walter, MC 2017, 'Safety and efficacy of olesoxime in patients with type 2 or non-ambulatory type 3 spinal muscular atrophy: a randomised, double-blind, placebo-controlled phase 2 trial', The Lancet Neurology, vol. 16, no. 7, pp. 513-522. https://doi.org/10.1016/S1474-4422(17)30085-6

Bertini, E. ; Dessaud, E. ; Mercuri, E. ; Muntoni, F. ; Kirschner, J. ; Reid, C. ; Lusakowska, A. ; Comi, G.P. ; Cuisset, J.-M. ; Abitbol, J.-L. ; Scherrer, B. ; Ducray, P.S. ; Buchbjerg, J. ; Vianna, E. ; van der Pol, W.L. ; Vuillerot, C. ; Blaettler, T. ; Fontoura, P. ; André, C. ; Bruno, C. ; Chabrol, B. ; Deconinck, N. ; Estournet, B. ; Fontaine-Carbonnel, S. ; Goemans, N. ; Gorni, K. ; Govoni, A. ; Guglieri, M. ; Lochmuller, H. ; Magri, F. ; Mayer, M. ; Müller-Felber, W. ; Rivier, F. ; Roper, H. ; Schara, U. ; Scoto, M. ; van den Berg, L. ; Vita, G. ; Walter, M.C. / Safety and efficacy of olesoxime in patients with type 2 or non-ambulatory type 3 spinal muscular atrophy: a randomised, double-blind, placebo-controlled phase 2 trial. In: The Lancet Neurology. 2017 ; Vol. 16, No. 7. pp. 513-522.

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AU - Mercuri, E.

AU - Muntoni, F.

AU - Kirschner, J.

AU - Reid, C.

AU - Lusakowska, A.

AU - Comi, G.P.

AU - Cuisset, J.-M.

AU - Abitbol, J.-L.

AU - Scherrer, B.

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AU - Chabrol, B.

AU - Deconinck, N.

AU - Estournet, B.

AU - Fontaine-Carbonnel, S.

AU - Goemans, N.

AU - Gorni, K.

AU - Govoni, A.

AU - Guglieri, M.

AU - Lochmuller, H.

AU - Magri, F.

AU - Mayer, M.

AU - Müller-Felber, W.

AU - Rivier, F.

AU - Roper, H.

AU - Schara, U.

AU - Scoto, M.

AU - van den Berg, L.

AU - Vita, G.

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