Onasemnogene abeparvovec gene therapy for symptomatic infantile-onset spinal muscular atrophy type 1 (STR1VE-EU): an open-label, single-arm, multicentre, phase 3 trial von Mercuri, Eugenio, Muntoni, Francesco, Baranello, Giovanni, Masson, Riccardo, Boespflug-Tanguy, Odile, Bruno, Claudio, Corti, Stefania, Daron, Aurore, Deconinck, Nicolas, Servais, Laurent, Straub, Volker, Ouyang, Haojun, Chand, Deepa, Tauscher-Wisniewski, Sitra, Mendonca, Nuno, Lavrov, Arseniy, Seferian, A., De Lucia, S., Tachibana, S., Jollet, A., Mouffak, S., Pedemonte, M., Brolatti, N., Morando, S., Vanlander, A., De Vos, E., Tahon, V., Govoni, A., Magri, F., Comi, G., Foa, M., Parente, V., Buscemi, L., Dal Farra, F., Schneider, O., Jonas, A., Defeldre, A.C., Pagliano, E., Zanin, R., Arnoldi, M.T., Schembri, V., Del Sole, M., Mandelli, A., Pera, M.C., Antonaci, L., Coratti, G., de Sanctis, R., Pane, M., Scoto, M., Groves, K., Edel, L., Abel, F., Van Ruiten, H., Lofra, R.M., Thompson, E.

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Therapeutic approaches for spinal muscular atrophy (SMA) von Scoto, M, Finkel, R S, Mercuri, E, Muntoni, F

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Mutations in the tail domain of DYNC1H1 cause dominant spinal: muscular atrophy von HARMS, M. B, ORI-MCKENNEY, K. M, MILLER, L. J, JANI-ACSADI, A, PESTRONK, A, SHY, M. E, MUNTONI, F, VALLEE, R. B, BALOH, R. H, SCOTO, M, TUCK, E. P, BELL, S, MA, D, MASI, S, ALLRED, P, AL-LOZI, M, REILLY, M. M

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Congenital myopathies – Clinical features and frequency of individual subtypes diagnosed over a 5-year period in the United Kingdom von Maggi, L, Scoto, M, Cirak, S, Robb, S.A, Klein, A, Lillis, S, Cullup, T, Feng, L, Manzur, A.Y, Sewry, C.A, Abbs, S, Jungbluth, H, Muntoni, F

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A new respiratory scoring system for evaluation of respiratory outcomes in children with spinal muscular atrophy type1 (SMA1) on SMN enhancing drugs von Edel, L, Grime, C, Robinson, V, Manzur, A, Abel, F, Munot, P, Ridout, D, Scoto, M, Muntoni, F, Chan, E

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Case study evaluating the effectiveness of intensive rehabilitation to improve function in a child with spinal muscular atrophy type 3 von Wadsworth, S., Abbott, L., Selby, V., Scoto, M., Main, M., Manzur, A.

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Involvement of the Heme-Oxygenase Pathway in the Antiallodynic and Antihyperalgesic Activity of Harpagophytum procumbens in Rats von Parenti, Carmela, Aricò, Giuseppina, Chiechio, Santina, Di Benedetto, Giulia, Parenti, Rosalba, Scoto, Giovanna M

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Supraspinal injection of Substance P attenuates allodynia and hyperalgesia in a rat model of inflammatory pain von Parenti, Carmela, Aricò, Giuseppina, Ronsisvalle, Giuseppe, Scoto, Giovanna M.

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The benzomorphan-based LP1 ligand is a suitable MOR/DOR agonist for chronic pain treatment von Pasquinucci, Lorella, Parenti, Carmela, Turnaturi, Rita, Aricò, Giuseppina, Marrazzo, Agostino, Prezzavento, Orazio, Ronsisvalle, Simone, Georgoussi, Zafiroula, Fourla, Danai-Dionysia, Scoto, Giovanna M., Ronsisvalle, Giuseppe

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SEPN1-related myopathies: Clinical course in a large cohort of patients von SCOTO, M, CIRAK, S, LONGMAN, C, CHOW, G, PANE, M, MAIN, M, HANNA, M. G, BUSHBY, K, SEWRY, C, ABBS, S, MERCURI, E, MUNTONI, F, MEIN, R, FENG, L, MANZUR, A. Y, ROBB, S, CHILDS, A.-M, QUINLIVAN, R. M, ROPER, H, JONES, D. H

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Antinociceptive profile of LP1, a non-peptide multitarget opioid ligand von Parenti, Carmela, Turnaturi, Rita, Aricò, Giuseppina, Marrazzo, Agostino, Prezzavento, Orazio, Ronsisvalle, Simone, Scoto, Giovanna M., Ronsisvalle, Giuseppe, Pasquinucci, Lorella

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Six minute walk test in type III spinal muscular atrophy: A 12 month longitudinal study von Mazzone, E, Bianco, F, Main, M, van den Hauwe, M, Ash, M, de Vries, R, Fagoaga Mata, J, Stein, S, De Sanctis, R, D’Amico, A, Palermo, C, Fanelli, L, Scoto, M.C, Mayhew, A, Eagle, M, Vigo, M, Febrer, A, Korinthenberg, R, de Visser, M, Bushby, K, Muntoni, F, Goemans, N, Sormani, M.P, Bertini, E, Pane, M, Mercuri, E

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Effects of intraplantar Nocistatin and (±)-J 113397 injections on nociceptive behavior in a rat model of inflammation von Scoto, Giovanna M., Aricò, Giuseppina, Ronsisvalle, Simone, Parenti, Carmela

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Hammersmith Functional Motor Scale and Motor Function Measure-20 in non ambulant SMA patients von Mazzone, E, De Sanctis, R, Fanelli, L, Bianco, F, Main, M, van den Hauwe, M, Ash, M, de Vries, R, Fagoaga Mata, J, Schaefer, K, D’Amico, A, Colia, G, Palermo, C, Scoto, M, Mayhew, A, Eagle, M, Servais, L, Vigo, M, Febrer, A, Korinthenberg, R, Jeukens, M, de Viesser, M, Totoescu, A, Voit, T, Bushby, K, Muntoni, F, Goemans, N, Bertini, E, Pane, M, Mercuri, E

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SMA REACH UK: Relationship between the Revised Hammersmith Scale (RHS) and Revised Upper Limb Module (RULM) in a UK cohort of patients with SMA type 2 and 3 von Tillmann, R, Milev, E, Ramsey, D, Main, M, Scoto, M, Muntoni, F

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Involvement of the Nociceptin/Orphanin FQ-NOP receptor system in the ventrolateral periaqueductal gray following mechanical allodynia in chronic pain von Scoto, Giovanna M., Aricò, Giuseppina, Iemolo, Attilio, Ronsisvalle, Simone, Parenti, Carmela

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Design and Synthesis of New Bifunctional Sigma‑1 Selective Ligands with Antioxidant Activity von Prezzavento, O, Arena, E, Parenti, C, Pasquinucci, L, Aricò, G, Scoto, G. M, Grancara, S, Toninello, A, Ronsisvalle, S

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Evaluation of N-substitution in 6,7-benzomorphan compounds von Pasquinucci, Lorella, Prezzavento, Orazio, Marrazzo, Agostino, Amata, Emanuele, Ronsisvalle, Simone, Georgoussi, Zafiroula, Fourla, Danai-Dionysia, Scoto, Giovanna M., Parenti, Carmela, Aricò, Giuseppina, Ronsisvalle, Giuseppe

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Six minute walk test in type III spinal muscular atrophy: A 12month longitudinal study von Mazzone, E., Bianco, F., Main, M., van den Hauwe, M., Ash, M., de Vries, R., Fagoaga Mata, J., Stein, S., De Sanctis, R., D’Amico, A., Palermo, C., Fanelli, L., Scoto, M.C., Mayhew, A., Eagle, M., Vigo, M., Febrer, A., Korinthenberg, R., de Visser, M., Bushby, K., Muntoni, F., Goemans, N., Sormani, M.P., Bertini, E., Pane, M., Mercuri, E.

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Microvascular defects secondary to SMN deficiency in spinal muscular atrophy von Zhou, H, Hong, Y, Scoto, M, Catapano, F, Aguti, S, Parson, S, Brogan, P, Muntoni, F

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