New hope for duchenne: targeted therapy now available for eligible patients
NCT ID NCT07250737
First seen Jun 27, 2026 · Last updated Jun 30, 2026 · Updated 2 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 and improve muscle function. Participants must be at least 2 years old and live in the US. This is not a cure, but aims to manage the condition long-term.
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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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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.
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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Nicklaus Children's Hospital
AVAILABLEMiami, Florida, 33155, 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 Pennsylvania
AVAILABLEPhiladelphia, Pennsylvania, 19104, United States
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University of Vermont Medical Center
AVAILABLEBurlington, Vermont, 05401, United States
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Washington University
AVAILABLESt Louis, Missouri, 63110, United States