New hope for duchenne: drug targets missing protein
NCT ID NCT04906460
First seen Oct 31, 2025 · Last updated May 14, 2026 · Updated 28 times
Summary
This study tests a drug called WVE-N531 in people with Duchenne muscular dystrophy (DMD), a genetic condition that causes muscle weakness. The drug aims to help the body produce a protein called dystrophin, which is missing in DMD. About 26 participants will receive the drug through an IV to see if it is safe and can improve muscle function. The study is still recruiting and includes multiple parts to gather more information.
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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
Conditions
Explore the condition pages connected to this study.