MICRO-DYSTROPHIN - Gene Transfer Study in Adolescents and Children with Duchenne

Frequently Asked Questions (FAQs) about this research:


What stage is this research?

  • This Phase 1/2 clinical trial is actively recruiting boys with Duchenne.


Where is this research being done and who is funding this research?

  • This research is being done by Solid Biosciences and its partners at the University of Missouri, the University of Washington, the University of Florida and Texas A&M University. Currently, the program is primarily funded by Solid Biosciences.  The program has also received funding from the National Institutes of Health (NIH) and the Department of Defense (DoD). Duchenne Research Fund, Duchenne UK and Joining Jack were initial investors in the project.


What is the goal or purpose of this research?

  • The goal of this research is to develop an adeno-associated virus (AAV) mediated Glossary Link gene therapy that enables the systemic delivery of a functional version of the dystrophin Glossary Link protein (microdystrophin) to the skeletal and cardiac muscles. This therapeutic approach will be studied for it potential to treat the majority of Duchenne patients, regardless of specific dystrophin Glossary Link mutation.


Who is eligible to participate in this trial?

  • Boys with Duchenne, ages 4 years - 17 years, are eligible to participate.  Both ambulatory and non-ambulatory boys may participate, but all must be on a stable dose of corticosteroids for at least 6 months.  Please visit ClinicalTrials.gov for the full list of inclusion and exclusion criteria.


Where is this clinical trial taking place?

  • The University of Florida in Gainesville, FL.


Where can I learn more about this research?

(Updated 12/18/2017)