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
Abou-Khalil, R., Yang, F., Mortreux, M., Lieu, S., Yu, Y. Y., Wurmser, M., Pereira, C., Relaix, F., Miclau, T., Marcucio, R. S., Colnot, C. :
Delayed bone regeneration is linked to chronic inflammation in murine muscular dystrophy J Bone Miner Res, 2014 ; 29 (2) : 304-15
Delayed bone regeneration is linked to chronic inflammation in murine muscular dystrophy J Bone Miner Res, 2014 ; 29 (2) : 304-15
Bombelli, F., Stojkovic, T., Dubourg, O., Echaniz-Laguna, A., Tardieu, S., Larcher, K., Amati-Bonneau, P., Latour, P., Vignal, O., Cazeneuve, C., Brice, A., Leguern, E. :
Charcot-Marie-Tooth Disease Type 2A: From Typical to Rare Phenotypic and Genotypic Features JAMA Neurol, 2014 ; 71 (8) : 1036-42
Charcot-Marie-Tooth Disease Type 2A: From Typical to Rare Phenotypic and Genotypic Features JAMA Neurol, 2014 ; 71 (8) : 1036-42
Fraysse, B., Vignaud, A., Fane, B., Schuh, M., Butler-Browne, G., Metzger, D., Ferry, A. :
Acute effect of androgens on maximal force-generating capacity and electrically evoked calcium transient in mouse skeletal muscles Steroids, 2014 ; 87C : 6-11
Acute effect of androgens on maximal force-generating capacity and electrically evoked calcium transient in mouse skeletal muscles Steroids, 2014 ; 87C : 6-11
Nazzal, D., Gradolatto, A., Truffault, F., Bismuth, J., Berrih-Aknin, S. :
Human thymus medullary epithelial cells promote regulatory T-cell generation by stimulating interleukin-2 production via ICOS ligand Cell Death Dis, 2014 ; 5 : e1420
Human thymus medullary epithelial cells promote regulatory T-cell generation by stimulating interleukin-2 production via ICOS ligand Cell Death Dis, 2014 ; 5 : e1420
