| Title |
Ongoing therapeutic trials and outcome measures for Duchenne muscular dystrophy
|
|---|---|
| Published in |
Cellular and Molecular Life Sciences, June 2013
|
| DOI | 10.1007/s00018-013-1396-z |
| Pubmed ID | |
| Authors |
Alessandra Govoni, Francesca Magri, Simona Brajkovic, Chiara Zanetta, Irene Faravelli, Stefania Corti, Nereo Bresolin, Giacomo P. Comi |
| Abstract |
Muscular dystrophy is a heterogeneous group of genetic disorders characterised by progressive muscle tissue degeneration. No effective treatment has been discovered for these diseases. Preclinical and clinical studies aimed at the development of new therapeutic approaches have been carried out, primarily in subjects affected with dystrophinopathies (Duchenne and Becker muscular dystrophy). In this review, we outline the current therapeutic approaches and past and ongoing clinical trials, highlighting both the advantages and limits of each one. The experimental designs of these trials were based on different rationales, including immunomodulation, readthrough strategies, exon skipping, gene therapy, and cell therapy. We also provide an overview of available outcome measures, focusing on their reliability in estimating meaningful clinical improvement in order to aid in the design of future trials. This perspective is extremely relevant to the field considering the recent development of novel therapeutic approaches that will result in an increasing number of clinical studies over the next few years. |
Mendeley readers
The data shown below were compiled from readership statistics for 118 Mendeley readers of this research output. Click here to see the associated Mendeley record.
Geographical breakdown
| Country | Count | As % |
|---|---|---|
| United Kingdom | 1 | <1% |
| United States | 1 | <1% |
| Portugal | 1 | <1% |
| Unknown | 115 | 97% |
Demographic breakdown
| Readers by professional status | Count | As % |
|---|---|---|
| Researcher | 20 | 17% |
| Student > Ph. D. Student | 19 | 16% |
| Student > Master | 18 | 15% |
| Student > Bachelor | 12 | 10% |
| Student > Postgraduate | 9 | 8% |
| Other | 19 | 16% |
| Unknown | 21 | 18% |
| Readers by discipline | Count | As % |
|---|---|---|
| Medicine and Dentistry | 30 | 25% |
| Agricultural and Biological Sciences | 25 | 21% |
| Biochemistry, Genetics and Molecular Biology | 10 | 8% |
| Nursing and Health Professions | 6 | 5% |
| Engineering | 5 | 4% |
| Other | 16 | 14% |
| Unknown | 26 | 22% |
Attention Score in Context
This research output has an Altmetric Attention Score of 7. 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 21 November 2023.
All research outputs
#4,863,939
of 23,794,258 outputs
Outputs from Cellular and Molecular Life Sciences
#921
of 4,151 outputs
Outputs of similar age
#40,690
of 198,650 outputs
Outputs of similar age from Cellular and Molecular Life Sciences
#7
of 41 outputs
Altmetric has tracked 23,794,258 research outputs across all sources so far. Compared to these this one has done well and is in the 79th percentile: it's in the top 25% of all research outputs ever tracked by Altmetric.
So far Altmetric has tracked 4,151 research outputs from this source. They typically receive a little more attention than average, with a mean Attention Score of 6.0. This one has done well, scoring higher than 84% 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 198,650 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 79% of its contemporaries.
We're also able to compare this research output to 41 others from the same source and published within six weeks on either side of this one. This one has done well, scoring higher than 82% of its contemporaries.