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Reviews

Review of Phase II and Phase III clinical trials for Duchenne muscular dystrophy

, MD, , MS PT & , MD
Pages 33-46 | Published online: 17 Dec 2012

Bibliography

  • Hoffman EP. Dystrophinopathies. In: Karpati G, Hilton-Jones D, Griggs R, editors. Disorders of voluntary muscle. Cambridge University Press; Cambridge: 2001. p. 385-432
  • Ciafaloni E, Fox DJ, Pandya S, Delayed diagnosis in duchenne muscular dystrophy: data from the Muscular Dystrophy Surveillance, Tracking, and Research Network (MD STARnet). J Pediatr 2009;155(3):380-5
  • Eagle M, Baudouin SV, Chandler C, Survival in Duchenne muscular dystrophy: improvements in life expectancy since 1967 and the impact of home nocturnal ventilation. Neuromuscular Disord 2002;12(10):926-9
  • Moxley RT III, Ashwal S, Pandya S, Practice parameter: corticosteroid treatment of Duchenne dystrophy: report of the Quality Standards Subcommittee of the American Academy of Neurology and the Practice Committee of the Child Neurology Society. Neurology 2005;64(1):13-20
  • Manzur AY, Kuntzer T, Pike M, Swan A. Glucocorticoid corticosteroids for Duchenne muscular dystrophy. Cochrane Database Syst Rev 2008(1):CD003725
  • Griggs RC, Moxley RT III, Mendell JR, Duchenne dystrophy: randomized, controlled trial of prednisone (18 months) and azathioprine (12 months). Neurology 1993;43(3 Pt 1):520-7
  • Mendell JR, Moxley RT, Griggs RC, Randomized, double-blind six-month trial of prednisone in Duchenne's muscular dystrophy. N Engl J Med 1989;320(24):1592-7
  • Fenichel GM, Florence JM, Pestronk A, Long-term benefit from prednisone therapy in Duchenne muscular dystrophy. Neurology 1991;41(12):1874-47
  • Griggs RC, Moxley RT, Mendell JR, Prednisone in duchenne dystrophy. A randomized, controlled trial defining the time course and dose response. Clinical Investigation of Duchenne Dystrophy Group. Arch Neurol 1991;48(4):383-8
  • Bonifati DM, Ruzza G, Bonometto P, A multicenter double-blind randomized trial of deflazacort versus prednisone in Duchenne muscular dystrophy. Muscle Nerve 2000;23(9):1344-7
  • Mesa LE, Dubrosvky AL, Corderi J, Steroids in Duchenne muscular dystrophy-deflazacort trial. Neuromuscular Disord 1991;1(4):261-6
  • Angelini C, Pegoraro E, Turella E, Deflazacort in Duchenne dystrophy: a study of long-term effect. Muscle Nerve 1994;17(4):386-91
  • Balaban B, Matthews DJ, Clayton GH, Carry T. Corticosteroid treatment and functional improvement in Duchenne muscular dystrophy: long-term effect. Am J Phys Med Rehabil 2005;84(11):843-50
  • Biggar WD, Harris VA, Eliasoph L, Alman B. Long-term benefits of deflazacort treatment for boys with Duchenne muscular dystrophy in their second decade. Neuromuscul Disord 2006;16(4):249-55
  • King WM, Ruttencutter R, Nagaraja HN, Orthopedic outcomes of long-term daily corticosteroid treatment in Duchenne muscular dystrophy. Neurology 2007;68(19):1607-13
  • Houde S, Filiatrault M, Fournier A, Deflazacort use in Duchenne muscular dystrophy: an 8-year follow up. Pediatr Neurol 2008;38(3):200-6
  • Merlini L, Gennari M, Malaspina E, Early corticosteroid treatment in 4 Duchenne muscular dystrophy patients: 14 year follow-up. Muscle Nerve 2012;45(6):796-802
  • Bushby K, Finkel R, Birnkrant DJ, Diagnosis and management of Duchenne muscular dystrophy, part 1: diagnosis, and pharmacological and psychosocial mangement. Lancet Neurol 2010;9(1):77-93
  • Bushby K, Finkel R, Birnkrant DJ, Diagnosis and management of Duchenne muscular dystrophy, part 2: implementation of multidisciplinary care. Lance Neurol 2010;9(2):177-89
  • Moxley RT III, Pandya S, Ciafaloni E, Change in natural history of Duchenne muscular dystrophy with long-term corticosteroid treatment: implications for management. J Child Neurol 2010;25(9):1116-29
  • McAdam LC, Mayo AL, Alman BA, Biggar WD. The Canadian experience with long-term deflazacort treatment in Duchenne muscular dystrophy. Acta Myologica 2012;31(1):16-20
  • Ahn AH, Kunkel LM. The structural and functional diversity of dystrophin. Nat Genet 1993;3(4):283-91
  • Aartsma-rus A, Van Deutekom JC, Fokkema IF, Entries in the Leiden Duchenne muscular dystrophy mutation database: an overview of mutation types and paradoxical cases that confirm the reading-frame rule. Muscle Nerve 2006;34(2):135-44
  • Monaco AP, Bertelson CJ, Liechti-Gallati S, An explanation for the phenotypic differences between patients bearing partial deletions of the DMD locus. Genomics 1988;2(1):90-5
  • Koenig M, Beggs AH, Moyer M, The molecular basis for Duchenne versus Becker muscular dystrophy: correlation of severity with type of deletion. Am J Hum Genet 1989;45(4):498-506
  • England SB, Nicholson LV, Johnson MA, Very mild muscular dystrophy associated with the deletion of 46% of dystrophin. Nature 1990;343(6254):180-2
  • Phelps SF, Hauser MA, Cole NM, Expression of full-length and truncated dystrophin mini-genes in transgenic mdx mice. Hum Mol Genet 1995;4(8):1251-8
  • Deburgrave N, Daoud F, Liense S, Protein- and mRNA-based phenotype-genotype correlations in DMD/BMD with point mutations and molecular basis for BMD with nonsense and frameshift mutations in the DMD gene. Hum Mutat 2007;28(2):183-95
  • Kirschner J, Schessl J, Schara U, Treatment of Duchenne muscular dystrophy with ciclosporin A: a randomised, double-blind, placebo-controlled multicentre trial. Lancet Neurol 2010;9(11):1053-9
  • Drachman DB, Tokya KV, Myer E. Prednisone in Duchenne muscular dystrophy. Lancet 1974;2(7894):1409-12
  • Escolar DM, Hache LP, Clemens PR, Randomized, blinded trial of weekend vs daily prednisone in Duchenne muscular dystrophy. Neurology 2011;77(5):444-52
  • Robert G. Finding the Optimum Regimen for Duchenne Muscular Dystrophy (FOR-DMD). In: clinicalTrials.gov [Internet] National Library of Medicine (US), Bethesda (MD); 2012. Available from: http://clinicaltrials.gov/ct2/show/NCT01603407?term=NCT01603407&rank=1: NCT01603407
  • Huizenga NA, Koper JW, De Lange P, A polymorphism in the glucocorticoid receptor gene may be associated with and increase sensitivity to glucocorticoids in vivo. J Clin Endocrinol Metab 1998;83(1):144-51
  • Reeves EK, Rayavarapu S, Damsker JM, Nagaraju K. Glucocorticoid anlogues: potential therapeutic alternatives for treating inflammatory muscle diseases. Endocr Metab Immune Disord Drug Targets 2012;12(1):95-103
  • Bonifati DM, Witchel SF, Ermani M, The glucocorticoid receptor N363S polymorphism and steroid response in Duchenne dystrophy. J Neurol Neurosurg Psychiatry 2006;77(10):1177-9
  • De Bosscher K, Beck IM, Haegeman G. Classic glucocorticoids versus non-steroidal glucocorticoid receptor modulators: survival of the fittest regulator of the immune system? Bran Behav Immun 2010;7):1035-42
  • Bello L, Piva L, Barp A, Importance of SPP1 genotype as a covariate in clinical trials in Duchenne muscular dystrophy. Neurology 2012;79(2):159-62
  • McPherron AC, Lawler AM, Lee SJ. Regulation of skeletal muscle mass in mice by a new TGF-beta superfamily member. Nature 1997;387(6628):83-90
  • McPherron AC, Lee SJ. Double muscling in cattle due to mutations in the myostatin gene. Proc Natl Acad Sci USA 1997;94(23):12457-61
  • Wagner KR, Fleckenstein JL, Amato AA, A phase I/IItrial of MYO-029 in adult subjects with muscular dystrophy. Ann Neurol 2008;63(5):561-71
  • Bish LT, Sleeper MM, Forbes SC, Long-term systemic myostatin inhibition via liver-targeted gene transfer in golden retriever muscular dystrophy. Hum Gene Ther 2011;22(12):1499-509
  • Schneyer AL, Sidis Y, Gulati A, Differential antagonism of activin, myostatin and growth and differentiation factor 11 by wild-type and mutant follistatin. Endocrinology 2008;149(9):4589-95
  • Nakatani M, Tekehara Y, Sugino H, Transgenic expression of a myostatin inhibitor derived from follistatin increases skeletal muscle mass and ameliorates dystrophic pathology in mdx mice. FASEB J 2008;22(2):477-87
  • Benabdallah BF, Bouchentouf M, Rousseau J, Inhibiting myostatin with follistatin improves the success of myoblast transplantation in dystrophic mice. Cell Transplant 2008;17(3):337-50
  • Jerry RM. Follistatin gene transfer to patients with becker muscular dystrophy and sporadic inclusion body myositis. In: clinicalTrials.gov [Internet] National Library of Medicine (US), Bethesda (MD); 2012. Available from: http://clinicaltrials.gov/ct2/show/NCT01519349?term=NCT01519349&rank=1: NCT01519349
  • Cadena SM, Tomkinson KN, Monell TE, Administration of soluble activin type IIB receptor promotes skeletal muscle growth independent of fiber type. J Appl Physiol 2010;109(3):635-42
  • Acceleron Pharma, Inc. A safety, tolerability, pharmacokinetic and pharmacodynamic study of ACE-031 in healthy postmenopausal women. In: ClinicalTrials.gov [Internet] National Library of Medicine (US), Bethesda (MD); Available from: http://clinicaltrials.gov/ct2/show/NCT00952887?term=ace-031&rank=1: NCT00952887
  • Acceleron Pharma, Inc. Study of ACE-031 in subjects with Duchenne muscular dystrophy. In: ClinicalTrials.gov [Internet] National Library of Medicine (US), Bethesda (MD); 2012. Available from: http://clinicaltrials.gov/ct2/show/NCT01099761?term=ace-031&rank=3 NCT01099761
  • Lee KH, Baek MY, Moon KY, Nitric oxide as a messenger molecule for myoblast fusion. J Biol Chem 1994;269(20):14371-4
  • Brenman JE, Chao DS, Xia H, Nitric oxide synthase complexed with dystrophin and absent from skeletal muscle sarcolemma in Duchenne muscular dystrophy. Cell 1995;82(5):743-52
  • Wehling M, Spencer MJ, Tidball JG. A nitric oxide synthase transgene ameliorates muscular dystrophy in mdx mice. J. Cell Biol 2001;155(1):123-31
  • Chang WJ, Iannaccone ST, Lau KS, Neuronal nitric oxide synthase and dystrophin-deficient muscular dystrophy. Proc Natl Acad Sci USA 1996;93(17):9142-7
  • Sander MB, Chavoshan B, Harris SA, Functional muscle ischemia in neuronal nitric oxide synthase deficient skeletal muscle of children with Duchenne muscular dystrophy. Proc Natl Acad Sci USA 2000;97(25):13818-23
  • Percival JM, Whitehead NP, Adams ME, Sildenafil reduces respiratory muscle weakness and fibrosis in the mdx mouse model of Duchenne muscular dystrophy. J Pathol 2012;228(1):77-87
  • Ron V. Tadalafil and sildenafil for duchenne muscular dystrophy. In: clinicaltrials.gov [Internet] National Library of Medicine (US), Bethesda (MD): 2012. Available from: http://clinicaltrials.gov/ct2/show/NCT01359670?term=sildenafil+duchenne&rank=2: NCT0135970
  • Escolar DM, Zimmerman A, Bertorini T, Pentoxifylline as a rescue treatment for DMD. A randomized double-blind clinical trial. Neurology 2012;78(12):904-13
  • Granchelli JA, Pollina C, Hudecki MS. Pre-clinical screening of drugs using the mdx mouse. Neuromuscul Disord 2000;10(4-5):235-9
  • Jorgensen JO, Moller L, Krag M, Effects of growth hormone on glucose and fat metabolism in human subjects. Endocrinol Metab Clin North Am 2007;36(1):75-87
  • Schertzer JD, van der Poel C, Shavlakadze T, Muscle-specific over expression of IGF-1 improves E-C coupling in skeletal muscle fibers from dystrophic mdx mice. Am J Physiol Cell Physiol 2008;294(1):C161-8
  • Ampong BN, Imamura M, Matsumiya T, Intracellular localization of dysferlin and its association with the dihydropyridine receptor. Acta Myol 2005;24(2):134-44
  • Rosenthal SM, Cheng ZQ. Opposing early and late effects of insulin-like growth factor I on differentiation and the cell cycle regulatory retinoblastoma protein in skeletal muscle. Proc Natl Acad Sci USA 1995;92(22):10307-10311
  • Engert JC, Berglund EB, Rosenthal N. Proliferation precedes differentiation in IGF-1-stimulated myogenesis. J Cell Biol 1996;135(2):431-40
  • Musaro A, McCullagh K, Paul A, Localized Igf-1 transgene expression sustains hypertrophy and regeneration in senescent skeletal muscle. Nat Genet 2001;27(2):195-200
  • Barton-Davis ER, Shorturma DI, Musaro A, Viral mediated expression of insulin-like growth factor I blocks the aging-related loss of skeletal muscle function. Proc Natl Acad Sci USA 1998;95(26):15603-7
  • Barton ER, Morris L, Musaro A, Muscle-specific expression of insulin-like growth factor I counters muscle decline in mdx mice. J Cell Biol 2002;157(1):137-48
  • Gregorevic P, Plant DR, Leeding KS, Improved contractile function of the mdx dystrophic mouse diaphragm muscle after insulin-like growth factor-I administration. Am J Pathol 2002;1(6):2263-72
  • Abmayr S, Gregorevic P, Allen JM, Chamberlain JS. Phenotypic improvement of dystrophic muscles by rAAV/microdystrophin vectors is augmented by Igf1 codelivery. Mol Ther 2005;12(3):441-50
  • Sacca F, Quarantelli M, Rinaldi C, A randomized controlled clinical trial of growth hormone in amyotrophic lateral sclerosis: clinical, neuroimaging, and hormonal results. J Neurol 2012;259(1):132-8
  • Sorenson EJ, Windbank AJ, Mandrekar JN, Subcutaneous IGF-1 is not beneficial in 2-year ALS trial. Neurology 2008;71(22):1770-5
  • Borasio GD, Robberecht W, Leigh PN, A placebo-controlled trial of insulin-like growth factor-I in amyotrophic lateral sclerosis. European ALS/IGF-1 Study Group. Neurology 1998;51(2):583-6
  • Heatwole CR, Eichinger KJ, Friedman DI, Open-label trial of recombinant human insulin-like growth factor 1/recombinant human insulin-like growth factor binding protein 3 in myotonic dystrophy type 1. Arch Neurol 2011;68(1):37-44
  • Vlachopapadopoulou E, Zachwieja JJ, Gertner JM, Metabolic and clinical response to recombinant human insulin-like growth factor 1 in myotonic dystrophy. J Clin Endocrinol Metab 1995;80(12):3715-23
  • Lai EC, Felice KJ, Festoff BW, Effect of recombinant human insulin-like growth factor-I on progression of ALS. A placebo controlled study. The North America ALS/IGF-1 Study Group. Neurology 1997;49(6):1621-30
  • Cohen P, Rogol AD, Howard CP, Insulin growth factor-based dosing of growth hormone therapy in children: a randomized, controlled study. J Clin Endocrinol Metab 2007;92(7):2480-6
  • Weissberger AJ, Anastasiadis AD, Strugess I, Recombinant human growth hormone treatment in elderly patients undergoing elective total hip replacement. Clin Endocrinol 2003;58(1):99-107
  • Rao MN, Mulligan K, Tai V, Effects of insulin-like growth factor (IGF)-1/IGF-binding protein-3 treatment on glucose metabolism and fat distribution in human immunodeficiency virus-infected patients with abdominal obesity and insulin resistance. J Clin Endocrinol Metab 2010;95(9):4361-6
  • Children's Hospital Medical Center, Cincinnati. Safety and efficacy study of IGF-1 in duchenne muscular dystrophy. In: ClinicalTrials.gov [Internet] National Library of Medicine (US), Bethesda (MD); 2012. Available from: http://clinicaltrials.gov/ct2/show/NCT01207908?term=IGF-1+duchenne&rank=1: NCT01207908
  • Novak F, Heyland DK, Avenell A, Glutamine supplementation in serious illness: a systemic review of the evidence. Crit Care Med 2002;30(9):2022-9
  • Hankard RG, Hammond D, Haymond MW, Darmaun D. Oral glutamine slows down whole body protein breakdown in Duchenne muscular dystrophy. Pediatr Res 1998;43(2):222-6
  • Hankard R, Mauras N, Hammond D, Is glutamine a ‘conditionally essential' amino acid in Duchenne muscular dystrophy? Clin Nutr 1999;18(6):365-9
  • Mok E, Letellier G, Cuisset JM, Lack of functional benefit with glutamine versus placebo in Duchenne muscular dystrophy: a randomized crossover trial. PLoS ONE 2009;4(5):e5448
  • Mendell JR, Griggs RC, Moxley RT III, Clinical investigation in Duchenne muscular dystrophy: IV. Double-blind controlled trial of leucine. Muscle Nerve 1984;7(7):535-41
  • Payne ET, Yasuda N, Bourgeois JM, Nutritional therapy improves function and complements corticosteroid intervention in mdx mice. Muscle Nerve 2006;33(1):66-77
  • Passaquin AC, Renard M, Kay L, Creatine supplementation reduces skeletal muscle degeneration and enhances mitochondrial function in mdx mice. Neuromuscul Disord 2002;12(2):174-82
  • Pulido SM, Passaquin AC, Leijendekker WJ, Creatine supplementation improves intracellular Ca2+ handling and survival in mdx skeletal muscle cells. FEBS Lett 1998;493(3):357-62
  • Tarnopolsky MA, Mahoney DJ, Vajsar J, Creatine monohydrate enhances strength and body composition in Duchenne muscular dystrophy. Neurology 2004;62(10):1771-7
  • Banerjee B, Sharma U, Balasubramanian K, Effect of creatine monohydrate in improving cellular energetics and muscle strength in ambulatory Duchenne muscular dystrophy patients: a randomized. placebo-controlled 31P MRS Study. Magn Reson Imaging 2010;28(5):698-707
  • Anderson WF, Gorini L, Breckenridge L. Role of ribosomes in streptomycin-activated suppression. Proc Natl Acad Sci USA 1965;54(4):1076-83
  • Davies J, Gilbert W, Gorini L. Streptomycin, suppression, and the code. Proc Natl Acad Sci USA 1964;51:883-90
  • Barton-Davis ER, Cordier L, Shotuma DI, Aminoglycoside antibiotics restore dystrophin function to skeletal muscles of mdx mice. J Clin Invest 1999;104(4):375-81
  • Wagner KR, Hamed S, Hadley DW, Gentamicin treatment of Duchenne and Becker muscular dystrophy due to nonsense mutations. Ann Neurol 2001;49(6):706-11
  • Politano L, Nigro G, Nigro V, Gentamicin administration in Duchenne patients with premature stop codon. Preliminary results. Acta Myol 2003;22(1):15-21
  • Malik V, Rodino-Klapac LR, Viollet L, Gentamicin-induced readthrough of stop codons in Duchenne muscular dystrophy. Ann Neurol 2010;67(6):771-80
  • Ghahramani Seno MM, Graham IR, Athanasopoulous T, RNAi-mediated knockdown of dystrophin expression in adult mice does not lead to overt muscular dystrophy pathology. Hum Mol Genet 2008;17:2622-32
  • Nudelman I, Rebibo-Sabbah A, Cherniavsky M. Development of novel aminoglycoside (NB54) with reduced toxicity and enhanced suppression of disease-causing premature stop mutations. J Med Chem 2009;52(9):2836-45
  • Lee HL, Dougherty JP. Pharmaceutical therapies to recode nonsense mutations in inherited diseases. Pharmacol Ther 2012;136(2):227-66
  • Welch EM, Barton ER, Zhuo J, PTC124 targets genetic disorders caused by nonsense mutations. Nature 2007;447:87-91
  • . PTC Therapeutics. Phase 2b Study of PTC124 in Duchenne/Becker Muscular Dystorphy (DMD/BMD). In: ClinicalTrials.gov [Internet] National Library of Medicine (US), Bethesda (MD); 2011. Available from: http://clinicaltrials.gov/ct2/show/NCT00592553?term=NCT00592553&rank=1: NCT00592553
  • PTC Therapeutics. Study of Ataluren for Previously Treated Patients With nmDBMD in the US. In: ClinicalTrials.gov [Internet] National Library of Medicine (US), Bethesda (MD); 2011. Available from: http://clinicaltrials.gov/ct2/show/NCT01247207?term=NCT01247207&rank=1: NCT01247207
  • Aartsma-Rus A, Janson AA, Kaman WE, Therapeutic antisense-induced exon skipping in cultured muscle cells from six different DMD patients. Hum Mol Genet 2003;12:907-14
  • Yokota T, Lu QL, Partridge T, Efficacy of systemic morpholino exon-skipping in duchenne dystrophy dogs. Ann Neurol 2009;65:667-76
  • Van Deutekom JC, Janson AA, Ginjaar LB, Local dystrophin restoration with antisense oligonucleotide PRO051. N Engl J Med 2007;357(26):2677-86
  • Goemans NM, Tullinius M, van den Akker JT, Systemic administration of PRO051 in Duchenne's muscular dystrophy. N Engl J Med 2011;364(16):1513-22
  • GlaxoSmithKline. A clinical study to assess the efficacy and safety of GSK2402968 in Subjects with Duchenne Muscular Dystrophy (DMD114044). In: ClinicalTrials.gov [Internet] National Library of Medicine (US), Bethesda (MD); 2010. Available from: http://clinicaltrials.gov/ct2/show/NCT01254019?term=NCT01254019&rank=1 NCT01254019
  • Cirak S, Feng L, Anthony K, Restoration of the dystrophin-associated glycoprotein complex after exon skipping therapy in Duchenne muscular dystrophy. Mol Ther 2012;20(2):462-7
  • Cirak S, Arechavala-Gomeza V, Guglieri M, Exon skipping and dystrophin restoration in patients with Duchenne muscular dystrophy after systemic phosphorodiamidate morpholino oligomer treatment: an open-label, phase 2, dose-escalation study. Lancet 2011;378(9791):595-605
  • Sarepta Therapeutics. Full 48-week Phase IIb data from eteplirsen study in Duchenne presented at World Muscle Society. [October 16, 2012]. Retrieved on October 26 2012. Availble from http://issuu.com/parentprojectmd/docs/mendell_eteplirsen_48_week_data_wms2012_final
  • Anthony K, Cirak S, Torelli S, Dystrophin quantification and clinical correlations in Becker muscular dystrophy: implications for clinical trials. Brain 2011;134(Pt 12):3547-59
  • Koenig M, Hoffman EP, Bertelson CJ, Complete cloning of the Duchenne muscular dystrophy (DMD) cDNA and preliminary genomic organization of the DMD gene in normal and affected individuals. Cell 1987;50(3):509-17
  • Scott JM, Li S, Harper SQ, Viral vectors for gene transfer of micro-, mini-, or full-length dystrophin. Neuromuscul Disord 2002(Suppl 1):S23-9
  • Wang B, Li J, Xiao X. Adeno-associated virus vector carrying human minidystrophin genes effectively ameliorates muscular dystrophy in the mdx mouse model. Proc Natl Acad Sci USA 2000;97(25):13614-719
  • Kornegay JN, Li J, Bogan JR, Widespread muscle expression of an AAV9 human mini-dystrophin vector after intravenous injection in neonatal dystrophin-deficient dogs. Mol Ther 2010;18(8):1501-18
  • Zhang Y, Duan D. Novel mini-dystrophin gene dual adeno-associated virus vectors restore neuronal nitric oxide synthase expression at the sarcolemma. Hum Gene Ther 2012;23(1):98-103
  • Mendell JR, Campbell K, Rodino-Klapac L, Dystrophin Immunity in Duchenne's Muscular Dystrophy. N Eng J Med 2010;363(15):1429-37
  • Florence J, Connolly A, Miller JP, Outcomes measure reliabiliity in non ambulatory boys and men with Duchenne Muscular Dystrophy (DMD): Results from the Muscular Dystrophy Association DMD clinical research network. Neurology 2012;78(Meeting Abstracts 1):P04.085
  • Lowes L, Alfano L, Viollet L, Moderate walking distances and velocity correlate with function comparable to 6 minute walk test. Neurology 2012;78(Meeting Abstracts 1):S49.005
  • Connolly A, Florence J, Cradock M, Motor and cognitive assessment of infants and young boys with duchenne muscular dystrophy; results from the muscular dystrophy association dmd clinical research center network. Neurology 2012;78(Meeting Abstracts 1):P04.084

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