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Editorial commentary
Daily or alternative, that is the question: steroid therapy for Duchenne muscular dystrophy patients
  1. Wen-Chen Liang1,
  2. Ichizo Nishino2,3
  1. 1Department of Pediatrics, Kaohsiung Medical University Hospital, Kaohsiung Medical University, Kaohsiung, Taiwan
  2. 2Department of Neuromuscular Research, National Institute of Neuroscience, Tokyo, Japan
  3. 3Department of Clinical Development, Translational Medical Center, National Center of Neurology and Psychiatry, Tokyo, Japan
  1. Correspondence to Dr Ichizo Nishino, Department of Neuromuscular Research, National Institute of Neuroscience, and Department of Clinical Development, Translational Medical Center, National Center of Neurology and Psychiatry, Tokyo, Japan; nishino{at}ncnp.go.jp

https://doi.org/10.1136/jnnp-2012-304230

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    Daily steroid use brings longer ambulation time but also more adverse effects, except for orthopaedic complications

    Duchenne muscular dystrophy (DMD) is an X-linked muscular dystrophy known to affect one in 3600 live male births and leads to progressive proximal muscle weakness in the first years, and orthopaedic, respiratory and cardiac complications in the teens. Typically the patients would lose the ability of ambulation at around the age of 10 years and decease in young adulthood if untreated. In recent years, corticosteroid treatment has been proved to prolong ambulation, improve quality of life and survival. Moreover, it has also shown efficacy in reducing the degree of scoliosis and improving cardiopulmonary function. …

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    Footnotes

    • Competing interests None.

    • Provenance and peer review Commissioned; externally peer reviewed.

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      Valeria Ricotti Deborah A Ridout Elaine Scott Ros Quinlivan Stephanie A Robb Adnan Y Manzur Francesco Muntoni on behalf of the NorthStar Clinical Network A Manzur F Muntoni S Robb R Quinlivan V Ricotti M Main Dubowitz Neuromuscular Centre, Great Ormond Street Hospital for Children NHS Trust, London K Bushby V Straub A Sarkozy M Guglieri E Strehle M Eagle A Mayhew H Roper H McMurchie A Childs K Pysden L Pallant S Spinty G Peachey A Shillington E Wraige H Jungbluth J Sheehan R Spahr I Hughes E Bateman C Cammiss T Willis L Groves N Emery P Baxter M Senior L Hartley B Parsons A Majumdar L Jenkins K Naismith A Keddie I Horrocks M Di Marco G Chow A Miah C de Goede N Thomas M Geary J Palmer C White K Greenfield E Scott
      Journal of Neurology, Neurosurgery & Psychiatry 2012; 84 698-705 Published Online First: 18 Dec 2012. doi: 10.1136/jnnp-2012-303902