In 1984, the word “Duchenne” entered my world. The progress we have made since then is astounding. For decades, we have united in our unwavering commitment to end Duchenne, and with recent achievements in this fight, we are experiencing a new reality where more individuals living with Duchenne and Becker have access to therapies than ever before. But there’s more that we need to learn and understand.

Recent breakthroughs in gene therapy have given us a foothold in this fight. We have moved from the theoretical to the possible in our ability to leverage gene therapy to deliver a modified version of the dystrophin protein to muscle. This is monumental progress, but we have much work ahead of us. We acknowledge that not any one therapy will end Duchenne—it will take a multi-faceted approach utilizing a combination of therapies. We need your help to take the next giant leap forward.

The path ahead is uncharted, but together we can advance our methods of studying approved and investigational therapies. This is why PPMD is launching the Dystrophinopathy Clinical Research Network (DCRN), a groundbreaking collaboration of researchers, clinicians, regulators, advocates, and citizen scientists that will leverage existing Duchenne clinical networks, infrastructure, and expertise to revolutionize how we develop therapies and combine them to slow progression and loss of function. I ask you to join the research evolution. Your gift to fund development of PPMD’s Dystrophinopathy Clinical Research Network by December 31st, will play a pivotal role in achieving our $500,000 goal to move the science further, faster.

Your donation to the DCRN isn’t just funding research—it’s providing hope to thousands of families. It’s giving people with Duchenne and Becker a redefined version of the future, and accelerating the pace of discovery from decades to years by utilizing innovative adaptive trial designs and incorporating novel data sharing methodologies.

Let’s advance the research evolution. Donate today and be an integral part of work that will impact this generation of patients through PPMD’s Dystrophinopathy Clinical Research Network.

We are at a pivotal moment in the fight to end Duchenne. With your support, we will create an ecosystem where collaboration, technology, and cross-disciplinary approaches continuously accelerate the pace of discovery and innovation.

Join us to advance the research evolution. New research for a new reality.

Sincerely,

Pat Furlong
Founding President & CEO