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Current and emerging treatment strategies for Duchenne muscular dystrophy

Overview of attention for article published in Neuropsychiatric Disease and Treatment, July 2016
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About this Attention Score

  • In the top 25% of all research outputs scored by Altmetric
  • High Attention Score compared to outputs of the same age (83rd percentile)
  • Good Attention Score compared to outputs of the same age and source (76th percentile)

Mentioned by

policy
1 policy source
twitter
2 tweeters
patent
4 patents

Citations

dimensions_citation
62 Dimensions

Readers on

mendeley
279 Mendeley
Title
Current and emerging treatment strategies for Duchenne muscular dystrophy
Published in
Neuropsychiatric Disease and Treatment, July 2016
DOI 10.2147/ndt.s93873
Pubmed ID
Authors

Jean Mah

Abstract

Duchenne muscular dystrophy (DMD) is the most common form of muscular dystrophy in childhood. It is caused by mutations of the DMD gene, leading to progressive muscle weakness, loss of independent ambulation by early teens, and premature death due to cardiorespiratory complications. The diagnosis can usually be made after careful review of the history and examination of affected boys presenting with developmental delay, proximal weakness, and elevated serum creatine kinase, plus confirmation by muscle biopsy or genetic testing. Precise characterization of the DMD mutation is important for genetic counseling and individualized treatment. Current standard of care includes the use of corticosteroids to prolong ambulation and to delay the onset of secondary complications. Early use of cardioprotective agents, noninvasive positive pressure ventilation, and other supportive strategies has improved the life expectancy and health-related quality of life for many young adults with DMD. New emerging treatment includes viral-mediated microdystrophin gene replacement, exon skipping to restore the reading frame, and nonsense suppression therapy to allow translation and production of a modified dystrophin protein. Other potential therapeutic targets involve upregulation of compensatory proteins, reduction of the inflammatory cascade, and enhancement of muscle regeneration. So far, data from DMD clinical trials have shown limited success in delaying disease progression; unforeseen obstacles included immune response against the generated mini-dystrophin, inconsistent evidence of dystrophin production in muscle biopsies, and failure to demonstrate a significant improvement in the primary outcome measure, as defined by the 6-minute walk test in some studies. The long-term safety and efficacy of emerging treatments will depend on the selection of appropriate clinical end points and sensitive biomarkers to detect meaningful changes in disease progression. Correction of the underlying mutations using new gene-editing technologies and corticosteroid analogs with better safety profiles offers renewed hope for many individuals with DMD and their families.

Twitter Demographics

The data shown below were collected from the profiles of 2 tweeters who shared this research output. Click here to find out more about how the information was compiled.

Mendeley readers

The data shown below were compiled from readership statistics for 279 Mendeley readers of this research output. Click here to see the associated Mendeley record.

Geographical breakdown

Country Count As %
Unknown 279 100%

Demographic breakdown

Readers by professional status Count As %
Student > Bachelor 61 22%
Student > Master 49 18%
Student > Ph. D. Student 44 16%
Researcher 31 11%
Student > Doctoral Student 13 5%
Other 31 11%
Unknown 50 18%
Readers by discipline Count As %
Biochemistry, Genetics and Molecular Biology 58 21%
Medicine and Dentistry 46 16%
Agricultural and Biological Sciences 32 11%
Pharmacology, Toxicology and Pharmaceutical Science 24 9%
Nursing and Health Professions 13 5%
Other 48 17%
Unknown 58 21%

Attention Score in Context

This research output has an Altmetric Attention Score of 10. This is our high-level measure of the quality and quantity of online attention that it has received. This Attention Score, as well as the ranking and number of research outputs shown below, was calculated when the research output was last mentioned on 29 December 2020.
All research outputs
#2,187,402
of 16,968,502 outputs
Outputs from Neuropsychiatric Disease and Treatment
#334
of 2,632 outputs
Outputs of similar age
#44,581
of 269,659 outputs
Outputs of similar age from Neuropsychiatric Disease and Treatment
#26
of 106 outputs
Altmetric has tracked 16,968,502 research outputs across all sources so far. Compared to these this one has done well and is in the 86th percentile: it's in the top 25% of all research outputs ever tracked by Altmetric.
So far Altmetric has tracked 2,632 research outputs from this source. They typically receive a little more attention than average, with a mean Attention Score of 6.7. This one has done well, scoring higher than 87% of its peers.
Older research outputs will score higher simply because they've had more time to accumulate mentions. To account for age we can compare this Altmetric Attention Score to the 269,659 tracked outputs that were published within six weeks on either side of this one in any source. This one has done well, scoring higher than 83% of its contemporaries.
We're also able to compare this research output to 106 others from the same source and published within six weeks on either side of this one. This one has done well, scoring higher than 76% of its contemporaries.