Cardiac involvement in the dystrophinopathies

Journal of Pediatric Rehabilitation Medicine

Volumn 1, Issue 3, 2008, Pages 211-223

  Russell, Mark W ^a   Townsend, DeWayne ^b   Metzger, Joseph M ^b  

^a UNIVERSITY OF MICHIGAN MEDICAL SCHOOL   (United States)

^b UNIVERSITY OF MINNESOTA MEDICAL SCHOOL   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

EID: 85013595533     PISSN: 18745393     EISSN: 18758894     Source Type: Journal    
DOI: None     Document Type: Article

References (94)

1. J. Alter, F. Lou, A. Rabinowitz, H. Yin, J. Rosenfeld, S.D. Wilton, T.A. Partridge and Q.L. Lu, Systemic delivery of morpholino oligonucleotide restores dystrophin expression bodywide and improves dystrophic pathology, Nat Med 12 (2006), 175-177.
2. American, A.o.P.S.o.C.a.C.S. Cardiovascular health supervision for individuals affected by Duchenne or Becker muscular dystrophy, Pediatrics 116 (2005), 1569-1573.
3. E.R. Barton-Davis, L. Cordier, D.I. Shoturma, S.E. Leland and H.L. Sweeney, Aminoglycoside antibiotics restore dystrophin function to skeletal muscles of mdx mice, J Clin Invest 104 (1999), 375-381.
4. D.M. Bers, Cardiac excitation-contraction coupling, Nature 415 (2002), 198-205.
5. N. Bhasin, R. Law, G. Liao, D. Safer, J. Ellmer, B.M. Discher, H.L. Sweeney and D.E. Discher, Molecular extensibility of mini-dystrophins and a dystrophin rod construct, J Mol Biol 352 (2005), 795-806.
6. L. Bidou, I. Hatin, N. Perez, V. Allamand, J.J. Panthier and J.P. Rousset, Premature stop codons involved in muscular dystrophies show a broad spectrum of readthrough efficiencies in response to gentamicin treatment, Gene Ther 11 (2004), 619-627.
7. W.D. Biggar, M. Gingras, D.L. Fehlings, V.A. Harris and C.A. Steele, Deflazacort treatment of Duchenne muscular dystrophy, J Pediatr 138 (2001), 45-50.
8. D.J. Birnkrant, H.B. Panitch, J.O. Benditt, L.J. Boitano, E.R. Carter, V.A. Cwik, J.D. Finder, S.T. Iannaccone, L.E. Jacobson, G.L. Kohn, E.K. Motoyama, R.T. Moxley, M.K. Schroth, G.D. Sharma and M.D. Sussman, American College of Chest Physicians consensus statement on the respiratory and related management of patients with Duchenne muscular dystrophy undergoing anesthesia or sedation, Chest 132 (2007), 1977-1986.
9. S. Bogdanovich, T.O. Krag, E.R. Barton, L.D. Morris, L.A. Whittemore, R.S. Ahima and T.S. Khurana, Functional improvement of dystrophic muscle by myostatin blockade, Nature 420 (2002), 418-421.
10. J.P. Bourke, Cardiac monitoring and treatment for children and adolescents with neuromuscular disorders, Dev Med Child Neurol 48 (2006), 164.
11. S.V. Brooks and J.A. Faulkner, Severity of contraction-induced injury is affected by velocity only during stretches of large strain, J Appl Physiol 91 (2001), 661-666.
12. K. Bushby, F. Muntoni, A. Urtizberea, R. Hughes and R. Griggs, Report on the 124th ENMC International Workshop. Treatment of Duchenne muscular dystrophy; defining the gold standards of management in the use of corticosteroids. 2-4 April 2004, Naarden, The Netherlands, Neuromuscul Disord 14 (2004), 526-534.
13. A.A. Chenard, H.M. Becane, F. Tertrain, J.M. de Kermadec and Y.A. Weiss, Ventricular arrhythmia in Duchenne muscular dystrophy: prevalence, significance and prognosis, Neuromuscul Disord 3 (1993), 201-206.
14. V. Chetboul, C. Escriou, D. Tessier, V. Richard, J.L. Pouchelon, H. Thibault, F. Lallemand, C. Thuillez, S. Blot and G. Derumeaux, Tissue Doppler imaging detects early asymptomatic myocardial abnormalities in a dog model of Duchenne’s cardiomyopathy, Eur Heart J 25 (2004), 1934-1939.
15. R.D. Cohn, C. van Erp, J.P. Habashi, A.A. Soleimani, E.C. Klein, M.T. Lisi, M. Gamradt, C.M. ap Rhys, T.M. Holm, B.L. Loeys, F. Ramirez, D.P. Judge, C.W. Ward and H.C. Dietz, Angiotensin II type 1 receptor blockade attenuates TGF-betainduced failure of muscle regeneration in multiple myopathic states, Nat Med 13 (2007), 204-210.
16. G. Corrado, A. Lissoni, S. Beretta, L. Terenghi, G. Tadeo, G. Foglia-Manzillo, L.M. Tagliagambe, M. Spata and M. Santarone, Prognostic value of electrocardiograms, ventricular late potentials, ventricular arrhythmias, and left ventricular systolic dysfunction in patients with Duchenne muscular dystrophy, Am J Cardiol 89 (2002), 838-841.
17. G.E. Crawford, J.A. Faulkner, R.H. Crosbie, K.P. Campbell, S.C. Froehner and J.S. Chamberlain, Assembly of the dystrophin-associated protein complex does not require the dystrophin COOH-terminal domain, J Cell Biol 150 (2000), 1399-1410.
18. L.H. Cripe, B.J. Barber, R.L. Spicer, B.L. Wong, N. Weidner, D.W. Benson and L.W. Markham, Outpatient continuous inotrope infusion as an adjunct to heart failure therapy in Duchenne muscular dystrophy, Neuromuscul Disord 16 (2006), 745-748.
19. D. Danilowicz, M. Rutkowski, D. Myung and D. Schively, Echocardiography in duchenne muscular dystrophy, Muscle Nerve 3 (1980), 298-303.
20. J.M. de Kermadec, H.M. Becane, A. Chenard, F. Tertrain and Y.Weiss, Prevalence of left ventricular systolic dysfunction in Duchenne muscular dystrophy: an echocardiographic study, Am Heart J 127 (1994), 618-623.
21. K.M. Dent, D.M. Dunn, A.C. von Niederhausern, A.T.Aoyagi, L. Kerr, M.B. Bromberg, K.J. Hart, T. Tuohy, S. White, J.T. den Dunnen, R.B. Weiss and K.M. Flanigan, K.M. Improved molecular diagnosis of dystrophinopathies in an unselected clinical cohort, Am J Med Genet A 134 (2005), 295-298.
22. D.B. Drachman, K.V. Toyka and E. Myer, Prednisone in Duchenne muscular dystrophy, Lancet 2 (1974), 1409-1412.
23. D. Duboc, C. Meune, G. Lerebours, J.Y. Devaux, G. Vaksmann and H.M. Becane, Effect of perindopril on the onset and progression of left ventricular dysfunction in Duchenne muscular dystrophy, J Am Coll Cardiol 45 (2005), 855-857.
24. P. Dunant, M.C. Walter, G. Karpati and H. Lochmuller, Gentamicin fails to increase dystrophin expression in dystrophindeficient muscle, Muscle Nerve 27 (2003), 624-627.
25. M. Eagle, S.V. Baudouin, C. Chandler, D.R. Giddings, R. Bullock and K. Bushby, Survival in Duchenne muscular dystrophy: improvements in life expectancy since 1967 and the impact of home nocturnal ventilation, Neuromuscul Disord 12 (2002), 926-929.
26. A. Emery, Genetic heterogeneity in Duchenne muscular dystrophy, Am J Med Genet 26 (1987), 235-236.
27. A. Emery, Muscular dystrophy-the facts, Neuromuscul Disord 5 (1995), 521.
28. A.E. Emery, X-linked muscular dystrophy with early contractures and cardiomyopathy (Emery-Dreifuss type), Clin Genet 32 (1987), 360-367.
29. A.E. Emery, Clinical and molecular studies in Duchenne muscular dystrophy, Prog Clin Biol Res 306 (1989), 15-28.
30. A.E. Emery, Population frequencies of inherited neuromuscular diseases - a world survey, Neuromuscul Disord 1 (1991), 19-29.
31. A.E. Emery, The muscular dystrophies, Lancet 359 (2002), 687-695.
32. A.E. Emery, Muscular dystrophy into the new millennium, Neuromuscul Disord 12 (2002), 343-349.
33. S.B. England, L.V. Nicholson, M.A. Johnson, S.M. Forrest, D.R. Love, E.E. Zubrzycka-Gaarn, D.E. Bulman, J.B. Harris and K.E. Davies, Very mild muscular dystrophy associated with the deletion of 46% of dystrophin, Nature 343 (1990), 180-182.
34. K.M. English and J.L. Gibbs, Cardiac monitoring and treatment for children and adolescents with neuromuscular disorders, Dev Med Child Neurol 48 (2006), 231-235.
35. J.M. Ervasti and K.P. Campbell, Dystrophin-associated glycoproteins: their possible roles in the pathogenesis of Duchenne muscular dystrophy, Mol Cell Biol Hum Dis Ser 3 (1993), 139-166.
36. J.M. Ervasti and K.P. Campbell, Dystrophin and the membrane skeleton, Curr Opin Cell Biol 5 (1993), 82-87.
37. J. Feng, J.Y. Yan, C.H. Buzin, S.S. Sommer and J.A. Towbin, Comprehensive mutation scanning of the dystrophin gene in patients with nonsyndromic X-linked dilated cardiomyopathy, J Am Coll Cardiol 40 (2002), 1120-1124.
38. J. Finsterer and C. Stollberger, The heart in human dystrophinopathies, Cardiology 99 (2003), 1-19.
39. K.M. Flanigan, A. von Niederhausern, D.M. Dunn, J. Alder, J.R. Mendell and R.B. Weiss, Rapid direct sequence analysis of the dystrophin gene. Am J Hum Genet 72 (2003), 931-939.
40. P.Y. Fong, P.R. Turner, W.F. Denetclaw and R.A. Steinhardt, Increased activity of calcium leak channels in myotubes of Duchenne human and mdx mouse origin, Science 250 (1990), 673-676.
41. G.S. Francis, Pathophysiology of chronic heart failure, Am J Med 110(Suppl 7A) (2001), 37S-46S.
42. A. Franco Jr. and J.B. Lansman, Calcium entry through stretchinactivated ion channels in mdx myotubes, Nature 344 (1990), 670-673.
43. K.A. Frankel and R.J. Rosser, The pathology of the heart in progressive muscular dystrophy: epimyocardial fibrosis, Hum Pathol 7 (1976), 375-386.
44. V. Giglio, V. Pasceri, L. Messano, F. Mangiola, L. Pasquini, A. Dello Russo, A. Damiani, M. Mirabella, G. Galluzzi, P. Tonali and E. Ricci, Ultrasound tissue characterization detects preclinical myocardial structural changes in children affected by Duchenne muscular dystrophy, J Am Coll Cardiol 42 (2003), 309-316.
45. R.M. Grady, H. Teng, M.C. Nichol, J.C. Cunningham, R.S. Wilkinson and J.R. Sanes, Skeletal and cardiac myopathies in mice lacking utrophin and dystrophin: a model for Duchenne muscular dystrophy, Cell 90 (1997), 729-738.
46. S.J. Haleen, R.E.Weishaar, R.W.Overhiser, R.F. Bousley, J.A. Keiser, S.R. Rapundalo and D.G. Taylor, Effects of quinapril, a new angiotensin converting enzyme inhibitor, on left ventricular failure and survival in the cardiomyopathic hamster. Hemodynamic, morphological, and biochemical correlates, Circ Res 68 (1991), 1302-1312.
47. S.A. Hamed and E.P. Hoffman, Automated sequence screening of the entire dystrophin cDNA in Duchenne dystrophy: point mutation detection, Am J Med Genet B Neuropsychiatr Genet 141 (2006), 44-50.
48. E.P. Hoffman, R.H. Brown Jr. and L.M. Kunkel, Dystrophin: the protein product of the Duchenne muscular dystrophy locus, Cell 51 (1987), 919-928.
49. S. Houde, M. Filiatrault, A. Fournier, J. Dube, S. D’Arcy, D. Berube, Y. Brousseau, G. Lapierre and M. Vanasse, Deflazacort use in duchenne muscular dystrophy: an 8-year follow-up, Pediatr Neurol 38 (2008), 200-206.
50. M.T. Howard, B.H. Shirts, L.M. Petros, K.M. Flanigan, R.F. Gesteland and J.F. Atkins, Sequence specificity of aminoglycoside-induced stop condon readthrough: potential implications for treatment of Duchenne muscular dystrophy, Ann Neurol 48 (2000), 164-169.
51. Y. Ishikawa, J.R. Bach and R. Minami, Cardioprotection for Duchenne’s muscular dystrophy, AmHeart J 137 (1999), 895-902.
52. J.L. Jefferies, B.W. Eidem, J.W. Belmont, W.J. Craigen, S.M. Ware, S.D. Fernbach, S.R. Neish, E.O. Smith and J.A.Towbin, Genetic predictors and remodeling of dilated cardiomyopathy in muscular dystrophy, Circulation 112 (2005), 2799-2804.
53. Y. Kamogawa, S. Biro, M. Maeda, M. Setoguchi, T. Hirakawa, H. Yoshida and C. Tei, Dystrophin-deficient myocardium is vulnerable to pressure overload in vivo, Cardiovasc Res 50 (2001), 509-515.
54. C. Kirchmann, D. Kececioglu, R. Korinthenberg and S. Dittrich, Echocardiographic and electrocardiographic findings of cardiomyopathy in Duchenne and Becker-Kiener muscular dystrophies, Pediatr Cardiol 26 (2005), 66-72.
55. G.A. Lanza, A. Dello Russo, V. Giglio, L. De Luca, L. Messano, C. Santini, E. Ricci, A. Damiani, G. Fumagalli, G. De Martino, F. Mangiola and F. Bellocci, Impairment of cardiac autonomic function in patients with Duchenne muscular dystrophy: relationship to myocardial and respiratory function, Am Heart J 141 (2001), 808-812.
56. D.R. Love, S.B. England, A. Speer, R.F. Marsden, J.F. Bloomfield, A.L. Roche, G.S. Cross, R.C. Mountford, T.J. Smith and K.E. Davies, Sequences of junction fragments in the deletionprone region of the dystrophin gene, Genomics 10 (1991), 57-67.
57. D.R. Love, S.M. Forrest, T.J. Smith, S. England, T. Flint, K.E. Davies and A. Speer, Molecular analysis of Duchenne and Becker muscular dystrophies, Br Med Bull 45 (1989), 659-680.
58. L.W. Markham, K. Kinnett, B.L.Wong, D. Woodrow Benson and L.H. Cripe, Corticosteroid treatment retards development of ventricular dysfunction in Duchenne muscular dystrophy, Neuromuscul Disord (2007), doi: 10.1016/j.nmd.2008.03.002
59. L.W. Markham, E.C. Michelfelder, W.L. Border, P.R. Khoury, R.L. Spicer, B.L. Wong, D.W. Benson and L.H. Cripe, Abnormalities of diastolic function precede dilated cardiomyopathy associated with Duchenne muscular dystrophy, J Am Soc Echocardiogr 19 (2006), 865-871.
60. L.W. Markham, R.L. Spicer, P.R. Khoury, B.L. Wong, K.D. Mathews and L.H. Cripe, Steroid therapy and cardiac function in Duchenne muscular dystrophy, Pediatr Cardiol 26 (2005), 768-771.
61. E.M. McNally, New approaches in the therapy of cardiomyopathy in muscular dystrophy, Annu Rev Med 58 (2007), 75-88.
62. P. Melacini, M. Fanin, A. Angelini, E. Pegoraro, U. Livi, G.A. Danieli, E.P. Hoffman, G. Thiene, S. Dalla Volta and C. Angelini, Cardiac transplantation in a Duchenne muscular dystrophy carrier, Neuromuscul Disord 8 (1998), 585-590.
63. J.R. Mendell, C.H. Buzin, J. Feng, J. Yan, C. Serrano, D.S. Sangani, C. Wall, T.W. Prior and S.S. Sommer, Diagnosis of Duchenne dystrophy by enhanced detection of small mutations, Neurology 57 (2001), 645-650.
64. P. Miura and B.J. Jasmin, Utrophin upregulation for treating Duchenne or Becker muscular dystrophy: how close are we? Trends Mol Med 12 (2006), 122-129.
65. T. Moriuchi, N. Kagawa, M. Mukoyama and K. Hizawa, Autopsy analyses of the muscular dystrophies, Tokushima J Exp Med 40 (1993), 83-93.
66. R. Ng, J.M. Metzger, D.R. Claflin and J.A. Faulkner, Poloxamer 188 reduces the contraction-induced force decline in lumbrical muscles from mdx mice, Am J Physiol 295 (2008), 146-150.
67. G. Nigro, L.I. Comi, L. Politano and R.J. Bain, The incidence and evolution of cardiomyopathy in Duchenne muscular dystrophy, Int J Cardiol 26 (1990), 271-277.
68. M.A. Nolan, O.D. Jones, R.L. Pedersen and H.M. Johnston, Cardiac assessment in childhood carriers of Duchenne and Becker muscular dystrophies, Neuromuscul Disord 13 (2003), 129-132.
69. E. Ozawa, Y. Mizuno, Y. Hagiwara, T. Sasaoka and M. Yoshida, Molecular and cell biology of the sarcoglycan complex, Muscle Nerve 32 (2005), 563-576.
70. M. Packer, A.J. Coats, M.B. Fowler, H.A. Katus, H. Krum, P. Mohacsi, J.L. Rouleau, M. Tendera, A. Castaigne, E.B. Roecker, M.K. Schultz and D.L. DeMets, Effect of carvedilol on survival in severe chronic heart failure, N Engl J Med 344 (2001), 1651-1658.
71. B.J. Petrof, J.B. Shrager, H.H. Stedman, A.M. Kelly and H.L. Sweeney, Dystrophin protects the sarcolemma from stresses developed during muscle contraction, Proc Natl Acad Sci USA 90 (1993), 3710-3714.
72. B. Pitt, F. Zannad, W.J. Remme, R. Cody, A. Castaigne, A. Perez, J. Palensky and J. Wittes, The effect of spironolactone on morbidity and mortality in patients with severe heart failure. Randomized Aldactone Evaluation Study Investigators, N Engl J Med 341 (1999), 709-717.
73. L. Politano, G. Nigro, V. Nigro, G. Piluso, S. Papparella, O. Paciello and L.I. Comi, Gentamicin administration in Duchenne patients with premature stop codon. Preliminary results, Acta Myol 22 (2003), 15-21.
74. L. Politano, V. Nigro, G. Nigro, V.R. Petretta, L. Passamano, S. Papparella, S. Di Somma and L.I. Comi, Development of cardiomyopathy in female carriers of Duchenne and Becker muscular dystrophies, Jama 275 (1996), 1335-1338.
75. T.W. Prior, C. Bartolo, D.K. Pearl, A.C. Papp, P.J. Snyder, M.S. Sedra, A.H. Burghes and J.R. Mendell, Spectrum of small mutations in the dystrophin coding region, Am J Hum Genet 57 (1995), 22-33.
76. R.M. Quinlivan and V. Dubowitz, Cardiac transplantation in Becker muscular dystrophy, Neuromuscul Disord 2 (1992), 165-167.
77. J.A. Rafael, G.A. Cox, K. Corrado, D. Jung, K.P. Campbell and J.S. Chamberlain, Forced expression of dystrophin deletion constructs reveals structure-function correlations, J Cell Biol 134 (1996), 93-102.
78. C. Ramaciotti, S.T. Iannaccone and W.A. Scott, Myocardial cell damage in Duchenne muscular dystrophy, Pediatr Cardiol 24 (2003), 503-506.
79. T. Ryoke, Y. Gu, L. Mao, M. Hongo, R.G. Clark, K.L. Peterson and J. Ross Jr., Progressive cardiac dysfunction and fibrosis in the cardiomyopathic hamster and effects of growth hormone and angiotensin-converting enzyme inhibition, Circulation 100 (1999), 1734-1743.
80. M. Sampaolesi, S. Blot, G. D’Antona, N. Granger, R. Tonlorenzi, A. Innocenzi, P. Mognol, J.L. Thibaud, B.G. Galvez, I. Barthelemy, L. Perani, S. Mantero, M. Guttinger, O. Pansarasa, C. Rinaldi, M.G. Cusella De Angelis, Y. Torrente, C. Bordignon, R. Bottinelli and G. Cossu, Mesoangioblast stem cells ameliorate muscle function in dystrophic dogs, Nature 444 (2006), 574-579.
81. M. Sander, B. Chavoshan, S.A. Harris, S.T. Iannaccone, J.T. Stull, G.D. Thomas and R.G. Victor, Functional muscle ischemia in neuronal nitric oxide synthase-deficient skeletal muscle of children with Duchenne muscular dystrophy, Proc Natl Acad Sci USA 97 (2000), 13818-13823.
82. K. Sasaki, K. Sakata, E. Kachi, S. Hirata, T. Ishihara and K. Ishikawa, Sequential changes in cardiac structure and function in patients with Duchenne type muscular dystrophy: a twodimensional echocardiographic study, Am Heart J 135 (1998), 937-944.
83. J.M. Scott, S. Li, S.Q. Harper, R. Welikson, D. Bourque, C. DelloRusso, S.D. Hauschka and J.S. Chamberlain, Viral vectors for gene transfer of micro-, mini-, or full-length dystrophin, Neuromuscul Disord 12(Suppl 1) (2002), S23-S29.
84. M.C. Silva, Z.M. Meira, J. Gurgel Giannetti, M.M. da Silva, A.F. Campos, M. Barbosa Mde, G.M. Starling Filho, A. Ferreira Rde, M. Zatz and C.E. Rochitte, Myocardial delayed enhancement by magnetic resonance imaging in patients with muscular dystrophy, J AmColl Cardiol 49 (2007), 1874-1879.
85. H.J. Spence, Y.J. Chen and S.J.Winder, Muscular dystrophies, the cytoskeleton and cell adhesion, Bioessays 24 (2002), 542-552.
86. A. Takenaka, M. Yokota, M. Iwase, K. Miyaguchi, H. Hayashi and H. Saito, Discrepancy between systolic and diastolic dysfunction of the left ventricle in patients with Duchenne muscular dystrophy, Eur Heart J 14 (1993), 669-676.
87. G.D. Thomas, M. Sander, K.S. Lau, P.L. Huang, J.T. Stull and R.G. Victor, Impaired metabolic modulation of alphaadrenergic vasoconstriction in dystrophin-deficient skeletal muscle, Proc Natl Acad Sci USA 95 (1998), 15090-15095.
88. G.D. Thomas, P.W. Shaul, I.S. Yuhanna, S.C. Froehner and M.E. Adams, Vasomodulation by skeletal muscle-derived nitric oxide requires alpha-syntrophin-mediated sarcolemmal localization of neuronal Nitric oxide synthase, Circ Res 92 (2003), 554-560.
89. D. Townsend, M.J. Blankinship, J.M. Allen, P. Gregorevic, J.S. Chamberlain and J.M. Metzger, Systemic administration of micro-dystrophin restores cardiac geometry and prevents dobutamine-induced cardiac pump failure, Mol Ther 15 (2007), 1086-1092.
90. J.C. van Deutekom, A.A. Janson, I.B. Ginjaar, W.S. Frankhuizen, A. Aartsma-Rus, M. Bremmer-Bout, J.T. den Dunnen, K. Koop, A.J. van der Kooi, N.M. Goemans, S.J. de Kimpe, P.F. Ekhart, E.H. Venneker, G.J. Platenburg, J.J. Verschuuren and G.J. van Ommen, Local dystrophin restoration with antisense oligonucleotide PRO051, N Engl J Med 357 (2007), 2677-2686.
91. K.R. Wagner, S. Hamed, D.W. Hadley, A.L. Gropman, A.H. Burstein, D.M. Escolar, E.P. Hoffman and K.H. Fischbeck, Gentamicin treatment of Duchenne and Becker muscular dystrophy due to nonsense mutations, Ann Neurol 49 (2001), 706-711.
92. E.M.Welch, E.R. Barton, J. Zhuo, Y. Tomizawa, W.J. Friesen, P. Trifillis, S. Paushkin, M. Patel, G.R. Trotta, S. Hwang, R.G. Wilde, G. Karp, J. Takasugi, G. Chen, S. Jones, H. Ren, Y.C. Moon, D. Corson, A.A. Turpoff, J.A. Campbell, M.M. Conn, A. Khan, N.G. Almstead, J. Hedrick, A. Mollin, N. Risher, M. Weetall, S. Yeh, A.A. Branstrom, J.M. Colacino, J. Babiak, W.D. Ju, S. Hirawat, V.J. Northcutt, L.L. Miller, P. Spatrick, F. He, M. Kawana, H. Feng, A. Jacobson, S.W. Peltz and H.L. Sweeney, PTC124 targets genetic disorders caused by nonsense mutations, Nature 447 (2007), 87-91.
93. S. Yasuda, D. Townsend, D.E. Michele, E.G. Favre, S.M. Day and J.M. Metzger, Dystrophic heart failure blocked by membrane sealant poloxamer, Nature 436 (2005), 1025-1029.
94. M. Yotsukura, K. Fujii, A. Katayama, Y. Tomono, H. Ando, K. Sakata, T. Ishihara and K. Ishikawa, Nine-year follow-up study of heart rate variability in patients with Duchenne-type progressive muscular dystrophy, Am Heart J 136 (1998), 289-296.