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Duchenne muscular dystrophy: hopes for the sesquicentenary

Edward Byrne, Andrew J Kornberg and Robert Kapsa
Med J Aust 2003; 179 (9): . || doi: 10.5694/j.1326-5377.2003.tb05648.x
Published online: 3 November 2003

We understand the molecular basis, and a cure may soon be possible


  • 1 Centre for Neuroscience, University of Melbourne, Carlton Sth, VIC.
  • 2 Department of Neurology, Royal Children’s Hospital, Parkville, VIC.
  • 3 Melbourne Neuromuscular Research Institute, Howard Florey Institute, Carlton Sth, VIC.


Correspondence: 

  • 1. Emery AE, Emery MLH. The history of a genetic disease. Duchenne muscular dystrophy or Meryon’s disease. London: Royal Society of Medicine Press, 1995.
  • 2. Duchenne (de Boulogne) fils. De la paralysie atrophique graisseuse de l’enfance. Archives Generales de Medecine 1864; 2 (6th ser., tome 4): 28-50, 184-209, 441-455.
  • 3. Monaco AP, Bertelson CJ, Middlesworth W, et al. Detection of deletions spanning the Duchenne muscular dystrophy locus using a tightly linked DNA segment. Nature 1985; 316: 842-845.
  • 4. Kunkel LM. Analysis of deletions from patients with Becker and Duchenne muscular dystrophy. Nature 1986; 322: 73-77.
  • 5. Koenig M, Monaco AP, Kunkel LM. The complete sequence of dystrophin predicts a rod shaped cytoskeletal protein. Cell 1988; 53: 219-226.
  • 6. Hoffman EP, Fischbeck KH, Brown RH, et al. Characterization of dystrophin in muscle biopsy specimens from patients with Duchenne’s or Becker’s muscular dystrophy. N Engl J Med 1988; 318: 1363-1368.
  • 7. Campbell KP, Kahl SD. Association of dystrophin and an integral membrane glycoprotein. Nature 1989; 338: 259-262.
  • 8. Kapsa R, Kornberg AJ, Byrne E. Novel therapies for Duchenne muscular dystrophy. Lancet Neurol 2003; 2: 299-310.
  • 9. Fassati A, Tedeschi S, Bordoni A, et al. Rapid direct diagnosis of deletions carriers of Duchenne and Becker muscular dystrophies. Lancet 1994; 344: 302-303.
  • 10. Griggs RC, Moxley RT 3rd, Mendell JR, et al. Duchenne dystrophy: randomized controlled trial of prednisone (18 months) and azathioprine (12 months). Neurology 1993; 43: 520-527.
  • 11. Luque ER. Segmental spinal instrumentation for correction of scoliosis. Clin Orthop 1982; 163: 192-198.
  • 12. Tremblay AP, Malouin F, Roy R, et al. Results of a triple blind clinical study of myoblast transplantations without immunosuppressive treatment in young boys with Duchenne muscular dystrophy. Cell Transplant 1993; 2: 99-112.

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