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
Publications (1184)
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
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
Hamel, Y., Mamoune, A., Mauvais, F. X., Habarou, F., Lallement, L., Romero, N. B., Ottolenghi, C., de Lonlay, P. :
Acute rhabdomyolysis and inflammation J Inherit Metab Dis, 2015 ; 38 (4) : 621-628
Acute rhabdomyolysis and inflammation J Inherit Metab Dis, 2015 ; 38 (4) : 621-628
Tuzun, E., Berrih-Aknin, S., Brenner, T., Kusner, L. L., Le Panse, R., Yang, H., Tzartos, S., Christadoss, P. :
Guidelines for standard preclinical experiments in the mouse model of myasthenia gravis induced by acetylcholine receptor immunization Exp Neurol, 2015 ; 270 : 11-17
Guidelines for standard preclinical experiments in the mouse model of myasthenia gravis induced by acetylcholine receptor immunization Exp Neurol, 2015 ; 270 : 11-17
de la Vaissiere, S., Toutain, A., Chene, M. A., Lagrue, E., Cantagrel, S., Provost, S., Eymard, B., Castelnau, P. :
Syndromes myastheniques congenitaux de l’enfant : strategies therapeutiques medicamenteuses Arch Pediatr, 2015 ; 22 (7) : 724-8
Syndromes myastheniques congenitaux de l’enfant : strategies therapeutiques medicamenteuses Arch Pediatr, 2015 ; 22 (7) : 724-8
Wirth, B., Barkats, M., Martinat, C., Sendtner, M., Gillingwater, T. H. :
Moving towards treatments for spinal muscular atrophy: hopes and limits Expert Opin Emerg Drugs, 2015 ; 20 (3) : 353-6
Moving towards treatments for spinal muscular atrophy: hopes and limits Expert Opin Emerg Drugs, 2015 ; 20 (3) : 353-6
