2025 Holiday Newsletter

Help Us Bring our AGU Kids One Step Closer to Treatment!

As we enter this Holiday Season, we’re reminded that every act of giving—large or small—moves our children with Aspartylglucosaminuria (AGU) closer to the life-changing treatment they urgently need. We invite you to stand with our families, researchers, and our incredible global community as we push toward a brighter, healthier future for kids with AGU 🙏

Why Your Partnership With Rare Trait Hope Matters

Rare Trait Hope and our sister charity, Rare Trait Hope Fund, are dedicated to raising funds to develop a gene therapy for families with children living with AGU, a devastating yet overlooked rare genetic disease.

Our mission is twofold: to accelerate treatment and to ensure no family walks this journey alone. YOU are a valued partner in this mission! 🤝

Through advocacy, research support, and global family coordination, we’re building a pathway to clinical trials and—ultimately— to safe, effective therapy for children whose time is limited without a treatment.

🌟 Your support fuels every milestone we reach! 🌟

Welcome Ollie – Our Newest AGU Warrior

Our global AGU community often says: “it takes many cities and many villages to get our children to treatment.” This year, one new village joined our fight—the loving community surrounding Ollie, a bright-eyed little boy from San Francisco who was diagnosed with AGU this fall.

In just two months, Ollie’s fearless mom and dad, family, and friends have raised over $30,000—through garage sales, church fundraiser, Ollie’s cousins selling homemade desserts and hand-poured candles, and even a heartfelt local fundraiser organized by his grandparents in Oaxaca, Mexico. Their determination reflects the resilience we see in families all over the world: people doing whatever it takes to give their children a chance at a healthier future.

Natural History Study Update – Over 40 Families Participating

This year, our sister charity, Rare Trait Hope Fund, helped us reach another major milestone with the launch of the AGU Registry and Natural History Study, a critical scientific effort that helps lay the groundwork for well-designed clinical trials. The registry is hosted on NORD’s IAMRARE® platform and sponsored by Rare Trait Hope Fund.

More than 40 families from Americas, Europe, Asia and Australia are now actively participating. Each family regularly uploads up-to-date information about their child’s symptoms, development, and health changes. This real-world data gives researchers a deeper understanding of how AGU progresses, helping refine safety benchmarks and clinical trial design. The dedication of these families is accelerating the path toward treatment more than ever before.

To learn more, visit: https://agu.iamrare.org. If you know a child with AGU, please encourage their parents to participate.

And Now, The BEST News of 2025!

A Historic Step Forward – FDA Drug Application Filing

We are thrilled to share a breakthrough years in the making: the drug application for our investigational gene therapy, DANAGALEX, is being filed with the U.S. Food and Drug Administration (FDA). Communication with the FDA is ongoing and clinical trial documentation continues to be submitted for review. This milestone is the culmination of years of manufacturing, safety testing, scientific reviews, and regulatory steps. 

This filing brings us closer than ever to securing FDA approval to begin the clinical trial for our children. For the first time, the treatment timeline feels real and within reach. Hope is no longer a distant vision—it’s coming clearly into view.

This Holiday Season – Help Us Cross the Finish Line

Thanks to you 🤗 we are closer than ever to delivering a life-changing therapy to kids with AGU. As we celebrate this historic step, we are also entering the most demanding and expensive phase of the journey: launching the clinical trial, supporting families, and sustaining essential research 👩‍⚕️👨‍⚕️🧑‍⚕️

Your donation can help:

🚀 Propel us through the final stages of trial readiness
🧭 Support families navigating the challenges of AGU
🔬 Strengthen international research partnerships
🙏 Bring transformative hope to children waiting for treatment

🌟 Every gift—monthly, one-time, stock, legacy, or corporate—moves us forward. Together, we can turn years of work into the moment our families have been fighting for. 🌟

Warm regards,