Goyenvalle, A., Griffith, G., Babbs, A., Andaloussi, S. E., Ezzat, K., Avril, A., Dugovic, B., Chaussenot, R., Ferry, A., Voit, T., Amthor, H., Buhr, C., Schurch, S., Wood, M. J., Davies, K. E., Vaillend, C., Leumann, C., Garcia, L. :
Functional correction in mouse models of muscular dystrophy using exon-skipping tricyclo-DNA oligomers
Nat Med, 2015 ; 21 (3) : 270-275
Publications (1184)
Tsonis, A I, Zisimopoulou, P, Lazaridis, K, Tzartos, J, Matsigkou, E, Zouvelou, V, Mantegazza, R, Antozzi, C, Andreetta, F, Evoli, A, Deymeer, F, Saruhan-Direskeneli, G, Durmus, H, Brenner, T, Vaknin, A, Berrih-Aknin, S, Behin, A, Sharshar, T, De Baets, M, Losen, M, Martinez-Martinez, P, Kleopa, K A, Zamba-Papanicolaou, E, Kyriakides, T, Kostera-Pruszczyk, A, Szczudlik, P, Szyluk, B, Lavrnic, D, Basta, I, Peric, S, Tallaksen, C, Maniaol, A, Casasnovas Pons, A, Pitha, J, JakubĂkova, M, Hanisch, F, Tzartos, S J :
MuSK autoantibodies in myasthenia gravis detected by cell based assay – a multinational study J Neuroimmunol, 2015 ; 284 : 10-17
MuSK autoantibodies in myasthenia gravis detected by cell based assay – a multinational study J Neuroimmunol, 2015 ; 284 : 10-17
Heslop, E., Csimma, C., Straub, V., McCall, J., Nagaraju, K., Wagner, K. R., Caizergues, D., Korinthenberg, R., Flanigan, K. M., Kaufmann, P., McNeil, E., Mendell, J., Hesterlee, S., Wells, D. J., Bushby, K., Laforet, P. :
The TREAT-NMD advisory committee for therapeutics (TACT): an innovative de-risking model to foster orphan drug development Orphanet J Rare Dis, 2015 ; 10 : 49
The TREAT-NMD advisory committee for therapeutics (TACT): an innovative de-risking model to foster orphan drug development Orphanet J Rare Dis, 2015 ; 10 : 49
Algalarrondo, V., Wahbi, K., Sebag, F., Gourdon, G., Beldjord, C., Azibi, K., Balse, E., Coulombe, A., Fischmeister, R., Eymard, B., Duboc, D., Hatem, S. N. :
Abnormal sodium current properties contribute to cardiac electrical and contractile dysfunction in a mouse model of myotonic dystrophy type 1 Neuromuscul Disord, 2015 ; 25 (4) : 308-320
Abnormal sodium current properties contribute to cardiac electrical and contractile dysfunction in a mouse model of myotonic dystrophy type 1 Neuromuscul Disord, 2015 ; 25 (4) : 308-320
Mauhin, W., Lidove, O., Masat, E., Mingozzi, F., Mariampillai, K., Ziza, J. M., Benveniste, O. :
Innate and Adaptive Immune Response in Fabry Disease JIMD Rep, 2015 ; 22 : 1-10
Innate and Adaptive Immune Response in Fabry Disease JIMD Rep, 2015 ; 22 : 1-10
Mingozzi, F., Buning, H. :
Adeno-Associated Viral Vectors at the Frontier between Tolerance and Immunity Front Immunol, 2015 ; 6 : 120
Adeno-Associated Viral Vectors at the Frontier between Tolerance and Immunity Front Immunol, 2015 ; 6 : 120
Wary, C., Azzabou, N., Giraudeau, C., Le Louer, J., Montus, M., Voit, T., Servais, L., Carlier, P. :
Quantitative NMRI and NMRS identify augmented disease progression after loss of ambulation in forearms of boys with Duchenne muscular dystrophy NMR Biomed, 2015 ; 28 (9) : 1150-62
Quantitative NMRI and NMRS identify augmented disease progression after loss of ambulation in forearms of boys with Duchenne muscular dystrophy NMR Biomed, 2015 ; 28 (9) : 1150-62
Allamand, V. :
Genetique Med Sci (Paris), 2015 ; 31 Spec No 3 : 28-9
Genetique Med Sci (Paris), 2015 ; 31 Spec No 3 : 28-9
Casar-Borota, O., Jacobsson, J., Libelius, R., Oldfors, C. H., Malfatti, E., Romero, N. B., Oldfors, A. :
A novel dynamin-2 gene mutation associated with a late-onset centronuclear myopathy with necklace fibres Neuromuscul Disord, 2015 ; 25 (4) : 345-348
A novel dynamin-2 gene mutation associated with a late-onset centronuclear myopathy with necklace fibres Neuromuscul Disord, 2015 ; 25 (4) : 345-348
Portilho, D. M., Alves, M. R., Kratassiouk, G., Roche, S., Magdinier, F., de Santana, E. C., Polesskaya, A., Harel-Bellan, A., Mouly, V., Savino, W., Butler-Browne, G., Dumonceaux, J. :
miRNA Expression in Control and FSHD Fetal Human Muscle Biopsies PLoS ONE, 2015 ; 10 (2) : e0116853
miRNA Expression in Control and FSHD Fetal Human Muscle Biopsies PLoS ONE, 2015 ; 10 (2) : e0116853
