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EXONDYS 51 for Duchenne Muscular Dystrophy
Study Summary
This trial will test the efficacy and safety of 3 drugs for treating boys with Duchenne Muscular Dystrophy who have a duplication of 1 of 3 exons. There will be weekly infusions and 2 muscle biopsies.
- Duchenne Muscular Dystrophy
Timeline
Treatment Details
Study Objectives
Outcome measures can provide a clearer picture of what you can expect from a treatment.Trial Design
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Who is running the clinical trial?
Frequently Asked Questions
What is the current enrollment threshold for this medical experiment?
"This investigation is not accepting any new patients at this time. It was listed on clinicaltrials.gov on the 18th of February 2020 and its most recent update came through on November 28th 2022. If you are searching for alternative trials, there are 86 studies concerning muscular dystrophies that are currently recruiting participants and 4 research projects related to Eteplirsen which have open enrollment periods."
Are there opportunities at this time for individuals to participate in the trial?
"Unfortunately, the current trial in question is no longer accepting patients. This was initially posted on February 18th 2020 and last edited November 28th 2022. Those searching for similar studies may be interested to learn that there are 86 muscular dystrophy trials recruiting participants as well as 4 open clinical trials involving Eteplirsen."
Is this the inaugural attempt to investigate such a phenomenon?
"Currently, there are 4 concurrent clinical studies centered around Eteplirsen in 28 different countries and 47 cities. This research was first initiated by Sarepta Therapeutics, Inc.'s phase 3 study with 222 participants back in 2016; since then 10 trials have been concluded."
Are there additional investigations exploring the effectiveness of Eteplirsen?
"Currently, there are 4 clinical studies in progress for Eteplirsen with 3 of them being Phase 3 trials. Although the majority of these tests are located in Milano and Mazowieckie, overall 157 medical institutions across the world have opened up their doors to this research."
Are there any potential adverse effects associated with taking Eteplirsen?
"Due to Eteplirsen's Phase 2 status, which indicates the presence of some safety data yet a lack of efficacy information, our team at Power has assigned this drug a score of 2."
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