Sibling concordance for clinical features of Duchenne and Becker muscular dystrophies

Sydney D Pettygrove, Zhenqiang Lu, Jennifer G. Andrews, F. John Meaney, Daniel W. Sheehan, Elinora T. Price, Deborah J. Fox, Shree Pandya, Lijing Ouyang, Susan D. Apkon, Zoe Powis, Christopher M Cunniff

Research output: Contribution to journalArticle

4 Citations (Scopus)

Abstract

Introduction: The correlation of markers of disease severity among brothers with Duchenne or Becker muscular dystrophy has implications for clinical guidance and clinical trials. Methods: Sibling pairs with Duchenne or Becker muscular dystrophy (n = 60) were compared for ages when they reached clinical milestones of disease progression, including ceased ambulation, scoliosis of ≥ 20°, and development of cardiomyopathy. Results: The median age at which younger brothers reached each milestone, compared with their older brothers ranged from 25 months younger for development of cardiomyopathy to 2 months older for ceased ambulation. For each additional month of ambulation by the older brother, the hazard of ceased ambulation by the younger brother decreased by 4%. Conclusions: The ages when siblings reach clinical milestones of disease vary widely between siblings. However, the time to ceased ambulation for older brothers predicts the time to ceased ambulation for their younger brothers.

Original languageEnglish (US)
Pages (from-to)814-821
Number of pages8
JournalMuscle and Nerve
Volume49
Issue number6
DOIs
StatePublished - 2014

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Duchenne Muscular Dystrophy
Walking
Cardiomyopathies
Scoliosis
Disease Progression
Clinical Trials

Keywords

  • Ambulation
  • Becker muscular dystrophy
  • Cardiomyopathy
  • Duchenne muscular dystrophy
  • Scoliosis
  • Sibling features

ASJC Scopus subject areas

  • Clinical Neurology
  • Cellular and Molecular Neuroscience
  • Physiology (medical)
  • Physiology

Cite this

Sibling concordance for clinical features of Duchenne and Becker muscular dystrophies. / Pettygrove, Sydney D; Lu, Zhenqiang; Andrews, Jennifer G.; Meaney, F. John; Sheehan, Daniel W.; Price, Elinora T.; Fox, Deborah J.; Pandya, Shree; Ouyang, Lijing; Apkon, Susan D.; Powis, Zoe; Cunniff, Christopher M.

In: Muscle and Nerve, Vol. 49, No. 6, 2014, p. 814-821.

Research output: Contribution to journalArticle

Pettygrove, SD, Lu, Z, Andrews, JG, Meaney, FJ, Sheehan, DW, Price, ET, Fox, DJ, Pandya, S, Ouyang, L, Apkon, SD, Powis, Z & Cunniff, CM 2014, 'Sibling concordance for clinical features of Duchenne and Becker muscular dystrophies', Muscle and Nerve, vol. 49, no. 6, pp. 814-821. https://doi.org/10.1002/mus.24078
Pettygrove, Sydney D ; Lu, Zhenqiang ; Andrews, Jennifer G. ; Meaney, F. John ; Sheehan, Daniel W. ; Price, Elinora T. ; Fox, Deborah J. ; Pandya, Shree ; Ouyang, Lijing ; Apkon, Susan D. ; Powis, Zoe ; Cunniff, Christopher M. / Sibling concordance for clinical features of Duchenne and Becker muscular dystrophies. In: Muscle and Nerve. 2014 ; Vol. 49, No. 6. pp. 814-821.
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AU - Sheehan, Daniel W.

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AU - Fox, Deborah J.

AU - Pandya, Shree

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