Experimental drug aims to restore muscle protein in boys with devastating muscle disease
NCT ID NCT04906460
Summary
This study is testing an investigational drug called WVE-N531 for boys and young men with Duchenne muscular dystrophy (DMD) who have a specific genetic mutation. The goal is to see if the drug is safe and if it can increase levels of a crucial muscle protein called dystrophin, which is missing in DMD. Participants receive the drug through an IV every few weeks and have muscle biopsies to measure protein changes and functional tests to track symptoms.
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Contacts and locations
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Study contacts
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Contact
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Arkansas Children's Hospital
RECRUITINGLittle Rock, Arkansas, 72202-3500, United States
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Istiklal Hospital/ Clinical Research Unit
RECRUITINGAmman, Jordan
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Oxford Children's Hospital, Oxford University Hospitals NHS Foundation Trust
RECRUITINGHeadington, Oxford, OX3 9DU, United Kingdom
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Rare Disease Research LLC
RECRUITINGAtlanta, Georgia, 30329, United States
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The Specialty Hospital (TSH)/ Advanced Clinical Center
RECRUITINGAmman, Jordan
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Conditions
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