New hope for duchenne: targeted therapy now available for eligible patients
NCT ID NCT07250737
First seen Jan 04, 2026 · Last updated May 15, 2026 · Updated 19 times
Summary
This program provides access to an experimental drug, AOC 1044, for people with Duchenne muscular dystrophy (DMD) whose genetic mutation can be treated by skipping exon 44. The goal is to help control the disease by allowing the body to produce a shorter but still functional dystrophin protein. Participants must be 6 or older, live in the US, and have the specific genetic mutation. This is not a cure, but aims to slow disease progression.
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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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UMass Memorial Health Care
AVAILABLEWorcester, Massachusetts, 01583, 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.