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 (1181)
Buyse, G. M., Voit, T., Schara, U., Straathof, C. S., D'Angelo, M. G., Bernert, G., Cuisset, J. M., Finkel, R. S., Goemans, N., McDonald, C. M., Rummey, C., Meier, T. :
Efficacy of idebenone on respiratory function in patients with Duchenne muscular dystrophy not using glucocorticoids (DELOS): a double-blind randomised placebo-controlled phase 3 trial Lancet, 2015 ; 385 (9979) : 1748-1757
Efficacy of idebenone on respiratory function in patients with Duchenne muscular dystrophy not using glucocorticoids (DELOS): a double-blind randomised placebo-controlled phase 3 trial Lancet, 2015 ; 385 (9979) : 1748-1757
Philippi, S, Lorain, S, Beley, C, Peccate, C, Précigout, G, Spuler, S, Garcia, L :
Dysferlin rescue by spliceosome-mediated pre-mRNA trans-splicing targeting introns harbouring weakly defined 3′ splice sites Hum Mol Genet, 2015 ; 24 (14) : 4049-4060
Dysferlin rescue by spliceosome-mediated pre-mRNA trans-splicing targeting introns harbouring weakly defined 3′ splice sites Hum Mol Genet, 2015 ; 24 (14) : 4049-4060
Benveniste, O. :
Myopathies inflammatoires Rev Prat, 2015 ; 65 (5) : 607-12
Myopathies inflammatoires Rev Prat, 2015 ; 65 (5) : 607-12
Kalman, B., Monge, C., Bigot, A., Mouly, V., Picart, C., Boudou, T. :
Engineering human 3D micromuscles with co-culture of fibroblasts and myoblasts Comput Methods Biomech Biomed Engin, 2015 ; 18 Suppl 1 : 1960-1
Engineering human 3D micromuscles with co-culture of fibroblasts and myoblasts Comput Methods Biomech Biomed Engin, 2015 ; 18 Suppl 1 : 1960-1
Urtizberea, J. A., Behin, A. :
Myopathie GNE Med Sci (Paris), 2015 ; 31 Spec No 3 : 20-7
Myopathie GNE Med Sci (Paris), 2015 ; 31 Spec No 3 : 20-7
Goudot, F. X., Wahbi, K., Aissou, L., Sorbets, E., Siam-Tsieu, V., Eymard, B., Themar Noel, C., Devaux, J. Y., Dessault, O., Duboc, D., Meune, C. :
Reduced inotropic reserve is predictive of further degradation in left ventricular ejection fraction in patients with Duchenne muscular dystrophy Eur J Heart Fail, 2015 ; 17 (2) : 177-81
Reduced inotropic reserve is predictive of further degradation in left ventricular ejection fraction in patients with Duchenne muscular dystrophy Eur J Heart Fail, 2015 ; 17 (2) : 177-81
Stenzel, W., Preusse, C., Allenbach, Y., Pehl, D., Junckerstorff, R., Heppner, F. L., Nolte, K., Aronica, E., Kana, V., Rushing, E., Schneider, U., Claeys, K. G., Benveniste, O., Weis, J., Goebel, H. H. :
Nuclear actin aggregation is a hallmark of anti-synthetase syndrome-induced dysimmune myopathy Neurology, 2015 ; 84 (13) : 1346-1354
Nuclear actin aggregation is a hallmark of anti-synthetase syndrome-induced dysimmune myopathy Neurology, 2015 ; 84 (13) : 1346-1354
Hogrel, J. Y., Barnouin, Y., Azzabou, N., Butler-Browne, G., Voit, T., Moraux, A., Leroux, G., Behin, A., McPhee, J. S., Carlier, P. G. :
NMR imaging estimates of muscle volume and intramuscular fat infiltration in the thigh: variations with muscle, gender, and age Age (Dordr), 2015 ; 37 (3) : 9798
NMR imaging estimates of muscle volume and intramuscular fat infiltration in the thigh: variations with muscle, gender, and age Age (Dordr), 2015 ; 37 (3) : 9798
Richardson, R. S., Wary, C., Walter Wray, D., Hoff, J., Rossiter, H., Layec, G., Carlier, P. G. :
MRS Evidence of Adequate O2 Supply in Human Skeletal Muscle at the Onset of Exercise (+ response : 2481-2) Med Sci Sports Exerc, 2015 ; 47 (11) : 2299-307
MRS Evidence of Adequate O2 Supply in Human Skeletal Muscle at the Onset of Exercise (+ response : 2481-2) Med Sci Sports Exerc, 2015 ; 47 (11) : 2299-307
