New hope for DMD: experimental drug opens to more patients
NCT ID NCT07250737
First seen Jan 04, 2026 · Last updated May 24, 2026 · Updated 22 times
Summary
This program provides access to an investigational drug, AOC 1044, for people with Duchenne muscular dystrophy (DMD) whose genetic mutation can be treated by skipping exon 44. It is for eligible patients aged 6 and older living in the US. The goal is to offer treatment while gathering more information on its effects.
Disclaimer
Read more
Show less
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.
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.
Get updates
Get notified about this study
Sign up to get updates when this study changes or when new studies for GENETIC DISEASES, INBORN are added.
Genom att skicka in godkänner du våra Användarvillkor
Contacts and locations
Show contact details
Enter your email to view the contact information for this study.
Genom att skicka in godkänner du våra Användarvillkor
Study contacts
-
Contact
Phone: •••-•••-•••• Email: •••••@•••••
Locations
-
Arkansas Children's Hospital
AVAILABLELittle Rock, Arkansas, 72202, United States
-
Gillette Children's
AVAILABLESaint Paul, Minnesota, 55101, United States
-
Nationwide Children's Hospital
AVAILABLEColumbus, Ohio, 43205, United States
-
Prisma Health-Midlands Children's Hospital
AVAILABLEColumbia, South Carolina, 29203, United States
-
Rare Disease Research
AVAILABLEKissimmee, Florida, 34746, United States
-
Rare Disease Research
AVAILABLEAtlanta, Georgia, 30329, United States
-
UMass Memorial Health Care
AVAILABLEWorcester, Massachusetts, 01583, United States
-
University of Kansas Medical Center
AVAILABLEKansas City, Kansas, 66103, United States
-
University of Vermont Medical Center
AVAILABLEBurlington, Vermont, 05401, United States
Conditions
Explore the condition pages connected to this study.