Dear Friends,
It’s hard to put into words just how meaningful the 2025 Family Conference and Research Workshop were—or how profoundly their impact will resonate across the entire RYR-1-Related Diseases (RYR-1-RD) community for years to come.
These events, and the organization’s work over the past decade, are at the heart of the mission of The RYR-1 Foundation—the only organization in the world solely dedicated to RYR-1-RD.
Major Accomplishments Over the Last Decade:
- Advocating for and with patients and families impacted by RYR-1-RD, exemplified through this year’s congressional advocacy campaign. Together with affected individuals and their loved ones, we engaged lawmakers to urge the continued funding of vital programs like Medical Assistance and the National Institutes of Health (NIH). This united effort emphasized the critical need for sustained investment in RYR-1-RD research and support.
- Bringing together almost 200 participants from twelve countries at the July 2025 Family Conference. While kids enjoyed The Funhouse Clubhouse and teens gathered in The Teen Spot, adults participated in a multitude of educational sessions—including expert panel and patient-led panel discussions, and one-on-one consults, among other offerings. Patients and families made lasting connections during meals and social activities.
- Convening a dynamic Research Workshop, which brought together the world’s leading scientists, healthcare providers, and early-career professionals—many engaging directly with patients whose stories were embedded into the event.
- Launching a new grant cycle (2025 – 2027), awarding $480,000 to four researchers pursuing innovative projects in gene therapies and the mechanisms of RYR-1-RD research.
- Providing education through free ongoing live and recorded webinars to the RYR-1-RD community. Recorded webinars are available on The RYR-1 Foundation’s website.
- Forming partnerships with organizations such as the Muscular Dystrophy Association (MDA) and AFM-Téléthon, both of which supported this year’s Research Workshop through grant funding.
- Expanding awareness through strategic collaboration, including meeting with biotech companies, and participating virtually in the American Society of Gene & Cell Therapy (ASGCT) conference—amplifying the voice of The RYR-1-Foundation and RYR-1-RD community.
The vital and vibrant work of The RYR-1 Foundation would not be possible without you—our global community of families, advocates, friends, and supporters. It’s with great appreciation that we share this recent progress and invite you to help us grow our impact.
To add to the momentum as we start a new decade, we ask for your continued support. Your donation expands resources, allows deeper scientific exploration, and gives greater hope to families living with RYR-1-RD. With your help, we can continue to transform our mission into action.
“For improved diagnosis and treatment, ongoing, up-to-date medical and scientific research is indispensable. But in the current climate of reduced federal support for such research, private donations have become even more important. The admirable research progress of The RYR-1 Foundation over the last 10 years demonstrates that continued private support can be tremendously successful in stimulating critically important medical and scientific research.” – Co-Chair of Research and Director Emeritus of The RYR-1 Foundation, Morton Goldberg, MD, FACS.
To make a gift, scan the QR code below or visit www.ryr1.org/donate. To explore planned giving options—including donations of securities, gifts through donor-advised funds (DAFs), wills, trusts, or other means—please feel free to contact me at lindsay@ryr1.org. Thank you for being part of this community and for helping to make our motto—Strength In Numbers—a powerful reality!
Best Wishes,
Lindsay Goldberg, BSN, RN
Executive Director, Patient Liaison & Co-Founder
The RYR-1 Foundation
