2024 Year in Review
2024 Year In Review
“The RYR-1 Foundation has transformed my life — I finally have hope.” – Individual affected by RYR-1-RD
Dear Friends,
As we celebrate the 10th anniversary of The RYR-1 Foundation, it is with great enthusiasm and gratitude that we share with you our accomplishments, not only over the past year, but also over the last decade.
When we founded The RYR-1 Foundation, we had the goal of serving the needs of individuals and families affected by RYR-1-Related Diseases (RYR-1-RD). Over the past decade, we have succeeded in building a vibrant community that includes affected families, healthcare providers, researchers, and donors from around the world.
As was the case 10 years ago, The RYR-1 Foundation remains the ONLY organization in the world dedicated solely to RYR-1-RD. Therefore, we continue to be a critical resource to not only affected individuals and families, but also researchers, healthcare providers, and biotechs. And, thanks to the generous support of donors, we have accomplished many significant milestones in pursuit of our mission.
Major Accomplishments Over the Last Decade:
- Raising more than $5.5 million, the majority of which came from individual donors
- Funding over $1.5 million in research grants, resulting in numerous articles published in peer-reviewed journals, the awarding of significant NIH grants, the creation of the first recessive RYR-1-RD mouse model, and, most importantly, advancing our understanding of RYR-1-RD and its implications for the development of potential therapies
- Hosting three International Family Conferences, attended by a total of 550 people from 10 countries
- Publishing the RYR-1-RD Clinical Care Guidelines (CCG), a free handbook for the RYR-1-RD community written in easily-comprehensible, non-technical language; the CCG has been translated into nine languages
- Providing webinars to the RYR-1-RD community; these are free and easily accessible via our website
- Conducting successful advocacy campaigns with leaders at NIH and in the US Congress to advance RYR-1-RD research
- Facilitating an RYR-1-RD clinical trial at NIH
- Expanding our Scientific Advisory Board, which now includes 15 internationally-recognized experts from eight different countries
- Leading a Patient-Led International Research Workshop, resulting in two recent peer-reviewed publications in an influential medical journal; this is in addition to many other research meetings we have hosted and sponsored in the United States and Europe
Plans for 2025:
- Awarding $500,000 in new research grants
- Hosting both the 2nd RYR-1-RD Patient-Led International Research Workshop and the 4th RYR-1 Foundation International Family Conference
- Launching an Advocacy Campaign focused on increasing federal funding for RYR-1-RD
And, we are just getting started! Although we continue to make great progress, we know that this is just the beginning of what we hope to achieve together with you. By growing partnerships, advocating for additional research funding, and advancing research, we are rapidly expanding our knowledge and understanding of RYR-1-RD. Of course, the ultimate goal for all of these efforts is to find an effective treatment or cure for the many individuals and families who struggle on a daily basis with RYR-1-RD.
As we celebrate the progress we’ve made together, we invite you to help shape the future by registering for the 2025 Family Conference, participating in one of our live webinars, joining our advocacy campaign, or reading our newsletter. Your participation is at the heart of everything we do.
Thank you for your continued involvement and being such an integral part of our community. With your engagement, we are closer than ever to turning our motto, “Strength in Numbers,” into reality!
If you have any questions, please reach out to Lindsay Goldberg or Michael Goldberg.
Once again, on behalf of the RYR-1-RD community and The RYR-1 Foundation, we sincerely thank you for your participation.
Best Wishes,
Lindsay Goldberg, BSN, RN
Executive Director & Co-Founder
The RYR-1 Foundation
Michael F. Goldberg, MD, MPH
Board President, Co-Chair of Research, & Co-Founder
The RYR-1 Foundation
