New hope for duchenne: experimental drug now available for eligible patients
NCT ID NCT07250737
First seen Jan 04, 2026 · Last updated Jun 13, 2026 · Updated 25 times
Summary
This program offers access to an experimental drug called AOC 1044 for people with Duchenne muscular dystrophy whose genetic mutation can be treated by skipping exon 44. It is for patients aged 6 and older who live in the US. The goal is to provide treatment while gathering more information about its effects.
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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
AVAILABLELittle Rock, Arkansas, 72202, United States
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Gillette Children's
AVAILABLESaint Paul, Minnesota, 55101, United States
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Nationwide Children's Hospital
AVAILABLEColumbus, Ohio, 43205, United States
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Prisma Health-Midlands Children's Hospital
AVAILABLEColumbia, South Carolina, 29203, United States
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Rare Disease Research
AVAILABLEKissimmee, Florida, 34746, United States
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Rare Disease Research
AVAILABLEAtlanta, Georgia, 30329, United States
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Rare Disease Research
AVAILABLEIselin, New Jersey, 08830, United States
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UMass Memorial Health Care
AVAILABLEWorcester, Massachusetts, 01583, United States
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University of Alabama
AVAILABLEBirmingham, Alabama, 35294, United States
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University of Kansas Medical Center
AVAILABLEKansas City, Kansas, 66103, United States
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University of Vermont Medical Center
AVAILABLEBurlington, Vermont, 05401, United States
Conditions
Explore the condition pages connected to this study.