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Drug Shows Promise in Treating a Type of Muscular Dystrophy in Boys

Drug Shows Promise in Treating a Type of Muscular Dystrophy in Boys

Our Review Summary

The approach to Duchenne muscular dystrophy described in the story is new and offers a potential approach to a host of genetic disorders.

Because the science is new and has not been studied in great detail, there are no guarantees that the approach will work in the long run and that side effects will not limit its usefulness.  This story did a reasonable job in describing a study based on information provided by the manufacturer and not yet published or even in abstract form.


Why This Matters

Reporting of clinical trial results should always be balanced and in context.  This is even more important when reporting about possible treatments for devastating diseases like Duchenne muscular dystrophy.


Does the story adequately discuss the costs of the intervention?

Not Applicable

Not applicable. The drug is in early stages of development and as such, identifying the potential cost is premature

Does the story adequately quantify the benefits of the treatment/test/product/procedure?


The story provided just enough caveats to the positive aspect of the study results.  “The study measured how far the boys could walk in six minutes. Those who received the higher dose walked an average of 21 meters farther after 48 weeks of treatment than at the beginning of the trial, a gain of about 5 percent. The boys in the placebo group walked 68 fewer meters at the end than at the beginning, a decline of about 17 percent.  One possible reason for skepticism is that two of the boys on a lower dose of the drug rapidly lost the ability to walk, even though the level of dystrophin in their muscles increased substantially.”

The story added a key piece of information at the end, “Eteplirsen actually turns off just one part of the dystrophin gene, known as exon 51, which contains a mutation that in some patients stops production of dystrophin. By skipping over this exon, muscle cells can produce an imperfect form of dystrophin that nonetheless is at least partly functional.

Does the story adequately explain/quantify the harms of the intervention?

Not Satisfactory

The story provided some limited information on what is known about the potential harms, “It is also unclear how long the effects of the drug would last or if safety issues would arise with longer treatment.”  “Eteplirsen, which Sarepta says has shown no side effects so far,…” 

The company’s press release noted: “The safety profile of eteplirsen was evaluated across all subjects through 48 weeks and there were no treatment-related adverse events, no serious adverse events, and no discontinuations. Furthermore, no clinically significant treatment-related changes were detected on any safety laboratory parameters, including several biomarkers for renal function. ” We think that this information should have been included in the story, as it would have provided more meaningful details to back up the sparse comments on harms that were published.  No, we don’t expect a story to read like a journal.  But we do draw a line between what we think is meaningful explanation to readers and what isn’t.

Does the story seem to grasp the quality of the evidence?


The story provided the reader with the caveats necessary to put the information into context.  “There are many caveats. The trial had only 12 patients, with only four receiving the high dose and four the placebo, and the data has not been reviewed by experts. It is also unclear how long the effects of the drug would last…” One possible reason for skepticism is that two of the boys on a lower dose of the drug rapidly lost the ability to walk, even though the level of dystrophin in their muscles increased substantially.

Does the story commit disease-mongering?


No disease mongering here

Does the story use independent sources and identify conflicts of interest?

Not Satisfactory

The story is based on a pre-abstract release of information from the company.  The study’s principal investigator, the company CEO and parents of children enrolled in the study are the only sources quoted in the story.  We think that an independent expert should have been quoted.

Does the story compare the new approach with existing alternatives?


The story noted that there is at least one other drug in development and that there is a rather ineffective treatment available in the form of steroids.

Does the story establish the availability of the treatment/test/product/procedure?


The story makes it clear that the drug is investigational and not available at the present.  It also provided some additional insight, “But Ms. McNary said Sarepta had refused to provide the drug to Max’s 13-year-old brother, Austin, who could not be in the trial because he already could not walk.”  “Mr. Garabedian said Sarepta did not have the money or the manufacturing capacity to make the drug available outside of clinical trials. “That would be the quickest path to insolvency,” he said.

Does the story establish the true novelty of the approach?


The story makes it clear that this is new and novel in the treatment of Duchenne muscular dystrophy

Does the story appear to rely solely or largely on a news release?


The story does not appear to rely on a press release.

Total Score: 7 of 9 Satisfactory


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