C. Bladen, K. Rafferty, V. Straub, S. Monges, A. Moresco et al., The TREAT-NMD Duchenne Muscular Dystrophy Registries: Conception, Design, and Utilization by Industry and Academia, Human Mutation, vol.115, issue.3-4, pp.1449-57, 2013.
DOI : 10.1159/000095920
URL : https://hal.archives-ouvertes.fr/hal-01681801

C. Bladen, D. Salgado, S. Monges, M. Foncuberta, K. Kekou et al., The TREAT-NMD DMD Global Database: Analysis of More than 7,000 Duchenne Muscular Dystrophy Mutations, Human Mutation, vol.447, issue.11, pp.395-402, 2015.
DOI : 10.1038/nature05756
URL : https://hal.archives-ouvertes.fr/hal-01681769

M. Pane, E. Mazzone, S. Sivo, M. Sormani, S. Messina et al., Long Term Natural History Data in Ambulant Boys with Duchenne Muscular Dystrophy: 36-Month Changes, PLoS ONE, vol.73, issue.10, p.108205, 2014.
DOI : 10.1371/journal.pone.0108205.s001

E. Henricson, R. Abresch, A. Cnaan, F. Hu, T. Duong et al., The cooperative international neuromuscular research group Duchenne natural history study: Glucocorticoid treatment preserves clinically meaningful functional milestones and reduces rate of disease progression as measured by manual muscle testing and othe, Muscle & Nerve, vol.4, issue.suppl, pp.55-67, 2013.
DOI : 10.1371/currents.RRN1297

J. Mendell, C. Shilling, N. Leslie, K. Flanigan, R. Dahhak et al., Evidence-based path to newborn screening for duchenne muscular dystrophy, Annals of Neurology, vol.10, issue.1, pp.304-317, 2012.
DOI : 10.1093/hmg/10.25.2851

W. Gowers and J. Taylor, A manual of diseases of the nervous system

V. Humbertclaude, D. Hamroun, K. Bezzou, C. Bérard, O. Boespflug-tanguy et al., Motor and respiratory heterogeneity in Duchenne patients: Implication for clinical trials, European Journal of Paediatric Neurology, vol.16, issue.2, pp.149-60, 2012.
DOI : 10.1016/j.ejpn.2011.07.001
URL : https://hal.archives-ouvertes.fr/hal-01681808

P. Baxter, Treatment of the heart in Duchenne muscular dystrophy, Developmental Medicine & Child Neurology, vol.45, issue.03, p.163, 2006.
DOI : 10.1161/CIRCULATIONAHA.104.528281

A. Emery, The muscular dystrophies. The Lancet, pp.687-95, 2002.

E. Hoffman, E. Reeves, J. Damsker, K. Nagaraju, J. Mccall et al., Novel Approaches to Corticosteroid Treatment in Duchenne Muscular Dystrophy. Physical Medicine and Rehabilitation Clinics of North America, pp.821-829, 2012.

T. Sejerson and K. Bushby, Standards of Care for Duchenne Muscular Dystrophy: Brief Treat-NMD Recommendations
DOI : 10.1007/978-90-481-2813-6_2

Z. Koeks, Clinical Outcomes in Duchenne Muscular Dystrophy In, Inherited Neuromuscular Diseases, pp.13-21, 2009.
URL : https://hal.archives-ouvertes.fr/hal-01681734

E. Landfeldt, P. Lindgren, C. Bell, C. Schmitt, M. Guglieri et al., Compliance to care guidelines for Duchenne muscular dystrophy in Italy, Neuromuscular Disorders, vol.28, issue.1, pp.63-72, 2015.
DOI : 10.1016/j.nmd.2016.09.018

B. Turnbull, The empirical distribution function with arbitrarily grouped, censored and truncated data, Journal of the Royal Statistical Society. Series B (Methodological), pp.290-95, 1976.

K. Bushby, R. Finkel, D. Birnkrant, L. Case, P. Clemens et al., Diagnosis and management of Duchenne muscular dystrophy, part 1: diagnosis, and pharmacological and psychosocial management, The Lancet Neurology, vol.9, issue.1, pp.77-93, 2010.
DOI : 10.1016/S1474-4422(09)70271-6

K. Bushby, R. Finkel, D. Birnkrant, L. Case, P. Clemens et al., Diagnosis and management of Duchenne muscular dystrophy, part 2: implementation of multidisciplinary care, The Lancet Neurology, vol.9, issue.2, pp.177-89, 2010.
DOI : 10.1016/S1474-4422(09)70272-8

A. Aartsma-rus, I. Fokkema, J. Verschuuren, I. Ginjaar, J. Van-deutekom et al., Theoretic applicability of antisense-mediated exon skipping for Duchenne muscular dystrophy mutations, Human Mutation, vol.7, issue.3, pp.293-302, 2009.
DOI : 10.1212/WNL.46.5.1347

A. Aartsma-rus, A. Janson, W. Kaman, M. Bremmer-bout, J. Den-dunnen et al., Therapeutic antisense-induced exon skipping in cultured muscle cells from six different DMD patients, Human Molecular Genetics, vol.12, issue.8, pp.907-921, 2003.
DOI : 10.1093/hmg/ddg100

G. Van-ommen, J. Van-deutekom, and A. Aartsma-rus, The therapeutic potential of antisense-mediated exon skipping, Curr Opin Mol Ther, vol.10, issue.2, pp.140-149, 2008.

G. Van-ommen and A. Aartsma-rus, Advances in therapeutic RNA-targeting, New Biotechnology, vol.30, issue.3, pp.299-301, 2013.
DOI : 10.1016/j.nbt.2013.01.005

K. Bushby, R. Finkel, B. Wong, R. Barohn, C. Campbell et al., Ataluren treatment of patients with nonsense mutation dystrophinopathy, Muscle & Nerve, vol.87, issue.4, pp.477-87, 2014.
DOI : 10.1038/ejhg.2009.122

D. Gloss, R. Moxley, S. Ashwal, and M. Oskoui, Practice guideline update summary: Corticosteroid treatment of Duchenne muscular dystrophy, Neurology, vol.86, issue.5, pp.465-72, 2016.
DOI : 10.1212/WNL.0000000000002337

L. Bello, L. Piva, A. Barp, A. Taglia, E. Picillo et al., Importance of SPP1 genotype as a covariate in clinical trials in Duchenne muscular dystrophy, Neurology, vol.79, issue.2, pp.159-62, 2012.
DOI : 10.1212/WNL.0b013e31825f04ea

J. Van-den-bergen, H. Ginjaar, E. Niks, A. Aartsma-rus, and J. Verschuuren, Prolonged ambulation in duchenne patients with a mutation amenable to exon 44 skipping, Journal of Neuromuscular Diseases, vol.1, issue.1, pp.91-95, 2014.

E. Matthews, R. Brassington, T. Kuntzer, F. Jichi, and A. Manzur, Corticosteroids for the treatment of Duchenne muscular dystrophy, Cochrane Database of Systematic Reviews, vol.269, issue.1, p.3725, 2016.
DOI : 10.1002/14651858.CD003725.pub2

Z. Rifai, S. Welle, R. Moxley, M. Lorenson, and R. Griggs, Effect of prednisone on protein metabolism in Duchenne dystrophy, American Journal of Physiology-Endocrinology and Metabolism, vol.86, issue.1, pp.67-74, 1995.
DOI : 10.1038/253192a0

S. Spuler and A. Engel, Unexpected sarcolemmal complement membrane attack complex deposits on nonnecrotic muscle fibers in muscular dystrophies, Neurology, vol.50, issue.1, pp.41-47, 1998.
DOI : 10.1212/WNL.50.1.41

R. Griggs, R. Moxley, J. Mendell, G. Fenichel, M. Brooke et al., Duchenne dystrophy: Randomized, controlled trial of prednisone (18 months) and azathioprine (12 months), Neurology, vol.43, issue.3, Part 1, pp.520-527, 1993.
DOI : 10.1212/WNL.43.3_Part_1.520

F. Pasquini, C. Guerin, D. Blake, K. Davies, G. Karpati et al., The effect of glucocorticoids on the accumulation of utrophin by cultured normal and dystrophic human skeletal muscle satellite cells, Neuromuscular Disorders, vol.5, issue.2, pp.105-119, 1995.
DOI : 10.1016/0960-8966(94)00042-8

R. Sklar, B. Jr, and R. , Methylprednisolone increases dystrophin levels by inhibiting myotube death during myogenesis of normal human muscle in vitro, Journal of the Neurological Sciences, vol.101, issue.1, pp.73-81, 1991.
DOI : 10.1016/0022-510X(91)90019-4

B. Barber, J. Andrews, Z. Lu, N. West, F. Meaney et al., Oral Corticosteroids and Onset of Cardiomyopathy in Duchenne Muscular Dystrophy, The Journal of Pediatrics, vol.163, issue.4, pp.1080-1084, 2013.
DOI : 10.1016/j.jpeds.2013.05.060

A. Smith, J. Koreska, and C. Moseley, Progression of scoliosis in Duchenne muscular dystrophy., The Journal of Bone & Joint Surgery, vol.71, issue.7, pp.1066-74, 1989.
DOI : 10.2106/00004623-198971070-00014

B. Alman, S. Raza, and W. Biggar, Steroid Treatment and the Development of Scoliosis in Males with Duchenne Muscular Dystrophy, The Journal of Bone & Joint Surgery, vol.86, issue.3, pp.519-543, 2004.
DOI : 10.2106/00004623-200403000-00009

Y. Yilmaz¨o, A. Karaduman, and H. Topalo?-glu, Prednisolone therapy in Duchenne muscular dystrophy prolongs ambulation and prevents scoliosis, European Journal of Neurology, vol.10, issue.1, pp.541-545, 2004.
DOI : 10.1002/(SICI)1097-4598(200004)23:4<521::AID-MUS10>3.0.CO;2-8

D. Townsend, S. Yasuda, J. Chamberlain, and J. Metzger, Cardiac Consequences to Skeletal Muscle-Centric Therapeutics for Duchenne Muscular Dystrophy, Trends in Cardiovascular Medicine, vol.19, issue.2, pp.49-54, 2009.
DOI : 10.1016/j.tcm.2009.04.006

J. Van-den-bergen, M. Hiller, S. Bohringer, L. Vijfhuizen, H. Ginjaar et al., variants, Journal of Neurology, Neurosurgery & Psychiatry, vol.6, issue.10, pp.1060-1065, 2015.
DOI : 10.1002/ana.10385

K. Flanigan, E. Ceco, K. Lamar, Y. Kaminoh, D. Dunn et al., genotype predicts age of ambulatory loss in duchenne muscular dystrophy, Annals of Neurology, vol.209, issue.1, pp.481-489, 2013.
DOI : 10.1016/j.atherosclerosis.2009.09.073

M. Pane, E. Mazzone, M. Sormani, S. Messina, G. Vita et al., 6 Minute Walk Test in Duchenne MD Patients with Different Mutations: 12 Month Changes, PLoS ONE, vol.48, issue.1, p.83400, 2014.
DOI : 10.1371/journal.pone.0083400.s002

A. Findlay, N. Wein, Y. Kaminoh, L. Taylor, D. Dunn et al., exon 45, Annals of Neurology, vol.1794, issue.pt 3, pp.668-74, 2015.
DOI : 10.1016/j.bbapap.2009.02.016
URL : https://hal.archives-ouvertes.fr/hal-01117017