New hope for duchenne: experimental drug aims to restore muscle protein
NCT ID NCT04906460
First seen Jun 24, 2026 · Last updated Jun 24, 2026
Summary
This study tests an experimental drug called WVE-N531 in 26 people with Duchenne muscular dystrophy who have a specific genetic flaw (exon 53 mutation). The drug is designed to help the body make a shortened but working version of dystrophin, a protein missing in DMD. The trial will check safety, how the drug moves through the body, and whether it increases dystrophin levels in muscle.
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This is a summary of
the original study
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Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
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Contacts and locations
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Study contacts
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
Locations
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Arkansas Children's Hospital
RECRUITINGLittle Rock, Arkansas, 72202-3500, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
Contact
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Istiklal Hospital/ Clinical Research Unit
RECRUITINGAmman, Jordan
Contact Email: •••••@•••••
Contact
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Oxford Children's Hospital, Oxford University Hospitals NHS Foundation Trust
RECRUITINGHeadington, Oxford, OX3 9DU, United Kingdom
Contact Phone: •••-•••-•••• Email: •••••@•••••
Contact
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Rare Disease Research LLC
RECRUITINGAtlanta, Georgia, 30329, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
Contact
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The Specialty Hospital (TSH)/ Advanced Clinical Center
RECRUITINGAmman, Jordan
Contact Email: •••••@•••••
Contact
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
Active substance
WVE-N531 (a drug designed to help the body produce a shortened but functional dystrophin protein)
What this could lead to
If successful, this could lead to a treatment that slows muscle decline and improves quality of life for people with Duchenne muscular dystrophy who have a specific genetic mutation.
What could go wrong
This is an early-phase trial with only 26 participants, so results may not apply to all patients. The drug may not produce enough dystrophin to make a real difference, and side effects are still being studied.
Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.