Hope for PDCD Foundation

05/13/2026

Definitely having some FOMO watching the American Society of Cell & Gene Therapy conference happening in Boston this week 🧬✨

We’re so excited to see friends of Hope for PDCD Foundation .podcast and and members of our research community with a poster on the E75A mouse model and future gene therapy approaches for PDCD.
It’s encouraging to see PDCD represented at a conference like ASGCT and to watch the research momentum continue to grow. 💜💚

05/05/2026

We are thrilled to share an exciting milestone for the PDCD community 💚💜

The NIH has officially shared a Notice of Award with the research team at for a grant to continue studying PDHA1 gene therapy. This funding will help deepen our understanding of emerging capsid technologies and move us closer to meaningful treatments, including the potential for gene therapy.

This achievement reflects the dedication and collaboration of an incredible team. We extend our heartfelt thanks to Dr. Steve Gray, Dr. Xin Chen, and Siyuan Hao, PhD Candidate and the entire team at UT Southwestern Medical Center.

Progress like this is only possible because of our community that shows up, believes in the mission and keeps pushing us forward.

04/30/2026

For children with PDCD, time is measured in milestones that may never be regained. Each delay carries lasting impact. The U.S. Food and Drug Administration must act with the urgency this disease demands.

04/29/2026

PDCD progresses quickly, often in early childhood. Families don’t have the luxury of time, and neither should the path forward. We call on the U.S. Food and Drug Administration to move with urgency.

04/28/2026

04/27/2026

Join Hope for PDCD Board Members Andrew Cieslinski and Dorian Minond on Sunday, May 3, at 1 pm ET, for an informative webinar on navigating Medicaid and special needs estate planning. Learn how these important tools can help protect your loved one with PDCD while providing greater security and peace of mind for your entire family.

This journey can be complex, but you don’t have to navigate it alone. Hear from fellow parents who are dedicated to supporting the PDCD community and empowering families with valuable guidance and resources. Together, we are stronger as we advocate for our loved ones. 💜💚

04/27/2026

This past weekend, the Hope for PDCD Board of Directors came together for a special hybrid retreat—some of us in person, others joining remotely, but all united by the same purpose. 💚 💜

We carved out intentional time to deepen our connections with one another, reflect on how far we’ve come, and reenergize for the work ahead. Most importantly, we rolled up our sleeves to strategize and lay the groundwork for the next chapter of Hope for PDCD.

Our focus is clear: advancing fundraising efforts, accelerating research, and strengthening family engagement so that no one in our community feels alone on this journey.
To our incredible board members—thank you for the time, heart, and dedication you bring to this mission. And to your spouses and family members, who made it possible to travel, show up, and fully engage—especially while coordinating care for medically complex children with PDCD—we see you, and we are so deeply grateful. Your support makes this work possible.

And to our broader PDCD community: everything we do is driven by you. We are honored to walk alongside you and remain deeply committed to building a stronger, more hopeful future together.

Onward. 💫

04/20/2026

The FDA has acknowledged that traditional trials are often impossible in ultra-rare diseases like PDCD. This is the moment to show how modern regulatory pathways can work for patients who cannot wait.

04/18/2026

When the physicians caring for children with PDCD speak collectively, it reflects urgency, experience, and deep understanding. The U.S. Food and Drug Administration has an opportunity to listen and act.

04/16/2026

PDCD is progressive and often fatal in early childhood. For families, time is not theoretical. Every delay can mean irreversible loss. The U.S. Food and Drug Administration must act with urgency.