December 19, 2017 / Clinical Trials,Research

Understanding Currently Recruiting Gene Therapy Studies

For the last many months, we have all been watching gene therapy make headlines around the world, across various diseases. As you know, earlier this year PPMD launched a Gene Therapy Initiative to help support the exploration of various gene therapy technologies in Duchenne.

Of particular note has been the progress made in micro-dystrophin gene therapy. There are currently three micro-dystrophin gene therapy trials in the early stages of recruitment. All three trials require a single infusion of a viral vector (the adeno-associated virus, or AAV) to deliver a shorter version of the dystrophin gene (a micro-dystrophin or mini-dystrophin).

In an effort to better understand the inclusion and exclusion criteria of these three trials, PPMD’s DuchenneConnect Registry team has compiled the following table highlighting the specifics of each trial so you can easily compare them and determine if one of these trials would be a good fit for your son.

(Click here to open this table in a new window.)

If you are interested in learning more about one of these trials, please visit to review all of the inclusion and exclusion criteria.  You may also contact the study coordinator at your site of interest.

In addition, read our new blog on pre-screening for antibodies which is required for all three of these gene therapy trials.

Additional Resources on Gene Therapy

Learn more about PPMD’s Gene Therapy Initiative, including links to our Gene Therapy Webinar Series.

Understanding Gene Therapy: The Screening Process

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