Flanigan, K. M., Voit, T., Rosales, X. Q., Servais, L., Kraus, J. E., Wardell, C., Morgan, A., Dorricott, S., Nakielny, J., Quarcoo, N., Liefaard, L., Drury, T., Campion, G., Wright, P. :
Pharmacokinetics and safety of single doses of drisapersen in non-ambulant subjects with Duchenne muscular dystrophy: Results of a double-blind randomized clinical trial
Neuromuscul Disord, 2014 ; 24 (1) : 16-24
Publications (1181)
Nathwani, A. C., Reiss, U. M., Tuddenham, E. G., Rosales, C., Chowdary, P., McIntosh, J., Della Peruta, M., Lheriteau, E., Patel, N., Raj, D., Riddell, A., Pie, J., Rangarajan, S., Bevan, D., Recht, M., Shen, Y. M., Halka, K. G., Basner-Tschakarjan, E., Mingozzi, F., High, K. A., Allay, J., Kay, M. A., Ng, C. Y., Zhou, J., Cancio, M., Morton, C. L., Gray, J. T., Srivastava, D., Nienhuis, A. W., Davidoff, A. M. :
Long-term safety and efficacy of factor IX gene therapy in hemophilia B N Engl J Med, 2014 ; 371 (21) : 1994-2004
Long-term safety and efficacy of factor IX gene therapy in hemophilia B N Engl J Med, 2014 ; 371 (21) : 1994-2004
Vassilopoulos, S., Gentil, C., Laine, J., Buclez, P. O., Franck, A., Ferry, A., Precigout, G., Roth, R., Heuser, J. E., Brodsky, F. M., Garcia, L., Bonne, G., Voit, T., Pietri-Rouxel, F., Bitoun, M. :
Actin scaffolding by clathrin heavy chain is required for skeletal muscle sarcomere organization J Cell Biol, 2014 ; 205 (3) : 377-93
Actin scaffolding by clathrin heavy chain is required for skeletal muscle sarcomere organization J Cell Biol, 2014 ; 205 (3) : 377-93
Avidan, N., Le Panse, R., Harbo, H. F., Bernasconi, P., Poulas, K., Ginzburg, E., Cavalcante, P., Colleoni, L., Baggi, F., Antozzi, C., Truffault, F., Horn-Saban, S., Poschel, S., Zagoriti, Z., Maniaol, A., Lie, B. A., Bernard, I., Saoudi, A., Illes, Z., Casasnovas Pons, C., Melms, A., Tzartos, S., Willcox, N., Kostera-Pruszczyk, A., Tallaksen, C., Mantegazza, R., Berrih-Aknin, S., Miller, A. :
VAV1 and BAFF, via NFkappaB pathway, are genetic risk factors for myasthenia gravis Ann Clin Transl Neurol, 2014 ; 1 (5) : 329-39
VAV1 and BAFF, via NFkappaB pathway, are genetic risk factors for myasthenia gravis Ann Clin Transl Neurol, 2014 ; 1 (5) : 329-39
Couvrat-Desvergnes, G., Masseau, A., Benveniste, O., Bruel, A., Hervier, B., Mussini, J. M., Buob, D., Hachulla, E., Remy, P., Azar, R., Namara, E. M., MacGregor, B., Daniel, L., Lacraz, A., De Broucker, T., Rouvier, P., Carli, P., Laville, M., Dantan, E., Hamidou, M., Moreau, A., Fakhouri, F. :
The spectrum of renal involvement in patients with inflammatory myopathies Medicine (Baltimore), 2014 ; 93 (1) : 33-41
The spectrum of renal involvement in patients with inflammatory myopathies Medicine (Baltimore), 2014 ; 93 (1) : 33-41
Hogrel, J. Y., Allenbach, Y., Canal, A., Leroux, G., Ollivier, G., Mariampillai, K., Servais, L., Herson, S., Decostre, V., Benveniste, O. :
Four-year longitudinal study of clinical and functional endpoints in sporadic inclusion body myositis: Implications for therapeutic trials Neuromuscul Disord, 2014 ; 24 (7) : 604-610
Four-year longitudinal study of clinical and functional endpoints in sporadic inclusion body myositis: Implications for therapeutic trials Neuromuscul Disord, 2014 ; 24 (7) : 604-610
Quijano-Roy, S., Rivier, F, Romero, N. B. :
Myopathies congénitales Lettre du Neurologue, 2014 ; 18 (7) : 263-266
Myopathies congénitales Lettre du Neurologue, 2014 ; 18 (7) : 263-266
Berrih-Aknin, S. :
Cortactin: A new target in autoimmune myositis and Myasthenia Gravis Autoimmun Rev, 2014 ; 13 (10) : 1001-2
Cortactin: A new target in autoimmune myositis and Myasthenia Gravis Autoimmun Rev, 2014 ; 13 (10) : 1001-2
Echaniz-Laguna, A., Dubourg, O., Carlier, P., Carlier, R. Y., Sabouraud, P., Pereon, Y., Chapon, F., Thauvin-Robinet, C., Laforet, P., Eymard, B., Latour, P., Stojkovic, T. :
Phenotypic spectrum and incidence of TRPV4 mutations in patients with inherited axonal neuropathy Neurology, 2014 ; 82 (21) : 1919-26
Phenotypic spectrum and incidence of TRPV4 mutations in patients with inherited axonal neuropathy Neurology, 2014 ; 82 (21) : 1919-26
Leshansky, L., Aberdam, D., Itskovitz-Eldor, J., Berrih-Aknin, S. :
Human Embryonic Stem Cells Prevent T-Cells Activation by Suppressing Dendritic Cells Function Via TGF-Beta Signaling Pathway Stem Cells, 2014 ; 32 (12) : 3137-49
Human Embryonic Stem Cells Prevent T-Cells Activation by Suppressing Dendritic Cells Function Via TGF-Beta Signaling Pathway Stem Cells, 2014 ; 32 (12) : 3137-49
