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Online self-report data for duchenne muscular dystrophy confirms natural history and can be used to assess for therapeutic benefits

PLoS Currents

Jan 31, 2015

Richard T WangStanley F Nelson

Abstract

To assess the utility of online patient self-report outcomes in a rare disease, we attempted to observe the effects of corticosteroids in delaying age at fulltime wheelchair use in Duchenne muscular dystrophy (DMD) using data from 1,057 males from DuchenneConnect, an online registry. Da...read more

Mentioned in this Paper

Study
Other medications
Ergocalciferol
steroid drug
Corticosteroids, topical for treatment of hemorrhoids and anal fissures
Vitamin D
Adrenal Cortex Hormones
Vitamin D [EPC]
DMD
Pharmaceutical Preparations
1
1
24
Paper Details
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Myopathies are genetic or acquired disorders of skeletal muscle that lead to varying degrees of weakness, atrophy, and exercise intolerance. Here are the latest discoveries pertaining to this disease.

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Corticosteroid treatment and functional improvement in Duchenne muscular dystrophy: long-term effect

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Online self-report data for duchenne muscular dystrophy confirms natural history and can be used to assess for therapeutic benefits

PLoS Currents

Jan 31, 2015

Richard T WangStanley F Nelson

PMID: 25635234

DOI: 10.1371/currents.md.e1e8f2be7c949f9ffe81ec6fca1cce6a

Abstract

To assess the utility of online patient self-report outcomes in a rare disease, we attempted to observe the effects of corticosteroids in delaying age at fulltime wheelchair use in Duchenne muscular dystrophy (DMD) using data from 1,057 males from DuchenneConnect, an online registry. Da...read more

Mentioned in this Paper

Study
Other medications
Ergocalciferol
steroid drug
Corticosteroids, topical for treatment of hemorrhoids and anal fissures
Vitamin D
Adrenal Cortex Hormones
Vitamin D [EPC]
DMD
Pharmaceutical Preparations
1
1
24

Similar Papers Found In These Feeds

Muscular Dystrophy

Muscular dystrophy refers to a range of muscle diseases caused by defects in muscle proteins, leading to death of the muscle cells, with loss of muscle tissue, and weakness. Here are the latest discoveries pertaining to this disease.

Myopathies

Myopathies are genetic or acquired disorders of skeletal muscle that lead to varying degrees of weakness, atrophy, and exercise intolerance. Here are the latest discoveries pertaining to this disease.

Related Papers

American Journal of Physical Medicine & Rehabilitation

Corticosteroid treatment and functional improvement in Duchenne muscular dystrophy: long-term effect

American Journal of Physical Medicine & RehabilitationOctober 26, 2005
Birol BalabanTerri Carry
Paper Details
References
  • References
  • Citations12
  • finger pointing at paper

    References currently unavailable

    We're still populating references for this paper, please check back later.
  • References
  • Citations12
12
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