| Title |
Eteplirsen in the treatment of Duchenne muscular dystrophy
|
|---|---|
| Published in |
Drug Design, Development and Therapy, February 2017
|
| DOI | 10.2147/dddt.s97635 |
| Pubmed ID | |
| Authors |
Kenji Rowel Q Lim, Rika Maruyama, Toshifumi Yokota |
| Abstract |
Duchenne muscular dystrophy is a fatal neuromuscular disorder affecting around one in 3,500-5,000 male births that is characterized by progressive muscular deterioration. It is inherited in an X-linked recessive fashion and is caused by loss-of-function mutations in the DMD gene coding for dystrophin, a cytoskeletal protein that stabilizes the plasma membrane of muscle fibers. In September 2016, the US Food and Drug Administration granted accelerated approval for eteplirsen (or Exondys 51), a drug that acts to promote dystrophin production by restoring the translational reading frame of DMD through specific skipping of exon 51 in defective gene variants. Eteplirsen is applicable for approximately 14% of patients with DMD mutations. This article extensively reviews and discusses the available information on eteplirsen to date, focusing on pharmacological, efficacy, safety, and tolerability data from preclinical and clinical trials. Issues faced by eteplirsen, particularly those relating to its efficacy, will be identified. Finally, the place of eteplirsen and exon skipping as a general therapeutic strategy in Duchenne muscular dystrophy treatment will be discussed. |
X Demographics
The data shown below were collected from the profiles of 12 X users who shared this research output. Click here to find out more about how the information was compiled.
Geographical breakdown
| Country | Count | As % |
|---|---|---|
| United States | 7 | 58% |
| Unknown | 5 | 42% |
Demographic breakdown
| Type | Count | As % |
|---|---|---|
| Members of the public | 9 | 75% |
| Scientists | 3 | 25% |
Mendeley readers
The data shown below were compiled from readership statistics for 711 Mendeley readers of this research output. Click here to see the associated Mendeley record.
Geographical breakdown
| Country | Count | As % |
|---|---|---|
| Unknown | 711 | 100% |
Demographic breakdown
| Readers by professional status | Count | As % |
|---|---|---|
| Student > Bachelor | 129 | 18% |
| Student > Ph. D. Student | 96 | 14% |
| Student > Master | 81 | 11% |
| Researcher | 64 | 9% |
| Other | 39 | 5% |
| Other | 95 | 13% |
| Unknown | 207 | 29% |
| Readers by discipline | Count | As % |
|---|---|---|
| Biochemistry, Genetics and Molecular Biology | 178 | 25% |
| Medicine and Dentistry | 75 | 11% |
| Agricultural and Biological Sciences | 54 | 8% |
| Pharmacology, Toxicology and Pharmaceutical Science | 44 | 6% |
| Chemistry | 37 | 5% |
| Other | 93 | 13% |
| Unknown | 230 | 32% |
Attention Score in Context
This research output has an Altmetric Attention Score of 55. 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 11 April 2023.
All research outputs
#775,776
of 25,382,440 outputs
Outputs from Drug Design, Development and Therapy
#39
of 2,268 outputs
Outputs of similar age
#16,906
of 424,972 outputs
Outputs of similar age from Drug Design, Development and Therapy
#2
of 45 outputs
Altmetric has tracked 25,382,440 research outputs across all sources so far. Compared to these this one has done particularly well and is in the 96th percentile: it's in the top 5% of all research outputs ever tracked by Altmetric.
So far Altmetric has tracked 2,268 research outputs from this source. They typically receive a little more attention than average, with a mean Attention Score of 7.1. This one has done particularly well, scoring higher than 98% 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 424,972 tracked outputs that were published within six weeks on either side of this one in any source. This one has done particularly well, scoring higher than 96% of its contemporaries.
We're also able to compare this research output to 45 others from the same source and published within six weeks on either side of this one. This one has done particularly well, scoring higher than 95% of its contemporaries.