Lengthy trials with variable results, based on endpoints such as the 6-minute walk test that can limit trial enrollment to ambulant individuals with Duchenne – this is today’s reality for clinical trials in this disease.

But progress is being made and PPMD is proud of our role in changing the clinical trial process. Last week,…

On September 28 and 29, I had the privilege of attending an in-person Wellstone Center meeting that Dr. Eric Olson and his Wellstone team at UT Southwestern Medical Center hosted in Dallas, TX. The breadth and depth of what the Wellstone Centers have worked on over the years was apparent. I was struck by the number of therapies in the clinic…

This fall, PPMD is presenting a gene therapy webinar series with companies and institutions who are developing therapies for Duchenne that are commonly referred to as gene therapy, including…