New hope for boys with devastating muscle disease
NCT ID NCT07250737
Summary
This program provides access to an investigational drug called AOC 1044 for eligible patients with Duchenne muscular dystrophy (DMD) who have a specific genetic mutation (amenable to exon 44 skipping). It is for patients aged 6 and older who are living in the US, including those who have completed a previous related study. The goal is to manage the disease by helping muscles produce a more functional protein.
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.
By submitting, you agree to our Terms of use
Contacts and locations
Show contact details
Enter your email to view the contact information for this study.
By submitting, you agree to our Terms of use
Study contacts
-
Contact
Phone: •••-•••-•••• Email: •••••@•••••
Locations
-
Gillette Children's
AVAILABLESaint Paul, Minnesota, 55101, United States
-
Rare Disease Research
AVAILABLEAtlanta, Georgia, 30329, United States
Conditions
Explore the condition pages connected to this study.