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Donavon Decker is an advocate, patient and the first person in the world to undergo gene therapy for any form of muscular dystrophy. Diagnosed with limb-girdle muscular dystrophy type 2D (LGMD2D), Donavon entered a safety trial in 1999 knowing it wouldn’t help him directly but hoped it would pave the way for future treatments, especially for his family members also affected by the disease.

Over the past 26 years, Donavon has become a leading voice for rare disease research, helping pass the MD-CARE Act, testifying before Congress, and serving on the National Institutes of Health Muscular Dystrophy Coordinating Committee. Despite losing three sisters to LGMD, he remains determined to accelerate progress. In 2023, he co-founded Angle Therapeutics to pursue non-viral gene therapies to allow for redosing and broader access.

Donavon continues to fight, not just for himself, but for every patient and family impacted by rare disease. His story is a testament to selfless advocacy and a call to action for faster, more equitable access to gene therapies.

In his own words, Donavon shares his LGMD2D patient journey and his path to working as an advocate.

In 1999, I became the first person in the world to undergo gene therapy for any form of muscular dystrophy.

It was a safety trial, and because it used an Adeno-Associated Virus (AAV), I knew I wouldn’t be able to receive it again. However, I pursued the trial to try and help others, especially my family.

I was born with Limb-Girdle Muscular Dystrophy type 2D (LGMD2D). Out of eight kids in my family, five of us were diagnosed with LGMD2D. One of my sisters who doesn’t have the disease has two daughters with another form, which is LGMD2I. The doctors told us it wasn’t supposed to happen that way, but it did.

This is why I’ve spent the last 26 years advocating for research, pushing for clinical trials, and trying to build a company to finally move this science forward.

I’ve lost three of my sisters to this disease. Watching them decline and die from something that still has no cure is a heartbreak that fuels me.

After I participated in the gene therapy trial in 1999 and told my story at the Muscular Dystrophy Association telethon hosted by Jerry Lewis, my sister June became the first person in the world to receive gene therapy via vascular delivery.

What we did wasn’t about our own condition. The data from those trials helped pave the way for the development of gene therapy for Spinal Muscular Atrophy. I’ve met families whose babies, who should have passed away by the age of two, are now seven years old and living full lives because of it.

That’s why I believe so strongly in gene therapy. I know it can work. This is why advocacy has become my life.

I’ve testified before the U.S. Senate, helped pass the MD-CARE Act in 2001, and served six years on the NIH Muscular Dystrophy Coordinating Committee. I’ve worked closely with scientists, policymakers, and patient advocates across the country. In 2025, I was honored with a Legacy Award from the Muscular Dystrophy Association for my work.

Despite all that, I’ve watched the research move too slowly. Some companies won’t invest in limb-girdle muscular dystrophy because they don’t see a profit in it, so our community and our families are left behind.

We can’t afford to keep waiting, and that’s why I decided to do something I never thought I’d be able to do.

In 2023, I co-founded a biotech company called Angle Therapeutics. Our mission is to develop a non-viral gene therapy platform to allow for redosing and overcome the cost and safety challenges involved with AAV-based therapies.

We don’t want to build everything from scratch because it would take too long. We’re looking to license an existing platform and run proof-of-concept studies in mice. If we like what we see, we’ll push forward with an exclusive license and begin the next steps toward a clinical trial.

We’ve already assembled a world-class scientific advisory board. The people we’re working with have some of the best minds in gene therapy, and I never imagined I’d have access to this kind of team.

If I could tell patients one thing, it’s if you’re eligible for a gene therapy trial, take it.

I’ve encouraged families to pursue trials even knowing they may not be perfect. Right now, it’s a chance, and that’s all we need to change the course of someone’s life.

We need to support clinical research, not just for ourselves, but for the entire community. The future of gene therapy depends on our ability to redose, scale treatments affordably, and to make them accessible to everyone, not just those with the most common diseases.

I’m 62 years old. I had a sister die when she was 65, and two other sisters die in their 50’s. I know time is running out for me, but even if I never benefit personally from this work, I’ll keep fighting. In a documentary about my journey, I state that one of the things I’d like to do one day is to be able to hug my wife, Kirsten. We’ve been married for 13 years now, and I can hug her, but she has to help me. Someday, I’d like to be able to give her a big old bear hug and wrap my arms around her. It would mean a lot to me, and I think it would mean a lot to her, too.

When you see a baby get a second chance at life, meet a mother whose child can now walk, breathe, and play, that changes you forever.

When I think about the families out there like mine, I know we can’t afford to give up, not when we know what’s possible.

We need the support, resources, and the belief that every life is worth saving.

I want to thank the Emily Whitehead Foundation for sharing my family’s story.

We receive messages from patients and families around the world with experiences similar to Donavon. We do whatever we can to be a resource for these families to help them get enrolled in a clinical trial or find a treatment center where they can access advanced therapies.

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