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Standards of Care for Duchenne Muscular Dystrophy: Brief Treat-NMD Recommendations

  • Thomas Sejerson
  • Kate BushbyEmail author
Chapter
Part of the Advances in Experimental Medicine and Biology book series (AEMB, volume 652)

Abstract

Care for patients with Duchenne muscular dystrophy (DMD) is poorly standardised. There are many interventions in different systems which are known to improve outcomes in DMD but these are not uniformly applied. This leads to inequality in access to treatment, as well as problems for planning controlled trials of future therapeutics. A worldwide effort is underway to generate care guidelines for DMD, which involves the Centre for Disease Control in the USA and the TREAT-NMD network of excellence for neuromuscular diseases in Europe. In advance of the full consensus document, TREAT-NMD has worked on the generation of brief standards of care for DMD, which are presented here and are available via the TREAT-NMD website (http://www.treat-nmd.eu). Guidelines are presented for diagnostics, neurological follow up, gastrointestinal and nutritional issues, respiratory and cardiac care as well as orthopaedics, rehabilitation, psychosocial interventions and oral care.

Keywords:

Duchenne muscular dystrophy Management Corticosteroids · Cardiomyopathy Respiratory support Rehabilitation Oral care 

Notes

Acknowledgements

TREAT-NMD is funded by the EU via FP6 contract number 036825. We are grateful for the participation of a number of experts in the generation of these care standards. These include:

Giovanni Nigro, Italy; Elizabeth Wraige, UK; Heinz Jungbluth, UK; John Bourke, UK; Thomas Sejersen, Sweden; Virginia Spehrs-Ciaffi, Switzerland; Gunna Sjoberg; Caroline Sewry, UK; Carina Wallgren-Pettersson, Finland; Francesco Muntoni, UK; H Ginjaar, Netherlands; Annie Aloysius, UK; Nathalie Goemans, Belgium; Trak Davies, UK; Adnan Manzur, UK; Birgit Steffensen, Denmark; Chiara Straathof, Netherlands; Corrado Angelini, Italy; Doug Biggar, Canada; Kate Bushby, UK; Rudolf Korinthenberg, Germany; Wendy King, USA; Elizabeth Vroom, Netherlands; Olé Nőrregård, Denmark; Benjamin Alman, Canada; Garaldine Edge, UK; Helena Saraste, Sweden; Corrado Angelini, Italy; Jos Hendriksen, Netherlands; Martin Chianani, UK; Anna-Karin Kroksmark, Sweden; Elaine Scott, UK; Jes Rahbek, Denmark; Michelle Eagle, UK; Anita Simonds, UK; Jonathan Finder, USA.

Further Reading

  1. 1.
    American Thoracic Society Consensus Conference (Finder JD, Chair). Respiratory care of the patient with Duchenne muscular dystrophy. Am J Crit Care Med 2004; 170:456–465.CrossRefGoogle Scholar
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    Bushby K, et al. 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 2004; 4:526–534.Google Scholar
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    Bushby K, et al. The multidisciplinary management of Duchenne muscular dystrophy. Curr Paediat 2005; 15:292–300.CrossRefGoogle Scholar
  4. 4.
    Duboc D, et al. Perindopril preventive treatment on mortality in Duchenne muscular dystrophy: 10 years’ follow-up. Am Heart J 2007; 154:596–602.CrossRefPubMedGoogle Scholar
  5. 5.
    Section on Cardiology and Cardiac Surgery. Cardiovascular health supervision for individuals affected by Duchenne or Becker muscular dystrophy. Pediatrics 2005; 116:1569–1573.CrossRefGoogle Scholar
  6. 6.
    Quinlivan R, et al.. Report of a Muscular Dystrophy Campaign Funded Workshop Birmingham, UK, January 16th 2004. Osteoporosis in Duchenne muscular dystrophy; its prevalence, treatment and prevention. Neuromuscul Disord 2005; 15:72–79.Google Scholar

Copyright information

© Springer Science+Business Media B.V. 2009

Authors and Affiliations

  1. 1.Astrid Lindgrens Barnsjukhus, Karolinska University HospitalStockholmSweden
  2. 2.Institute of Human GeneticsInternational Centre for LifeCentral Parkway, Newcastle upon TyneUK

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