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.

We deeply appreciate your support, whether by participating in our programs, attending our Family Conferences, joining one of our webinars, taking part in our patient advocacy efforts, and by being part of our RYR-1-Related Diseases (RYR-1-RD) community. We are also deeply grateful for those of you that have provided a donation to us. Your generosity, no matter the size of your gift, helps us get closer to finding a cure.

When we founded The RYR-1 Foundation, we had the goal of serving the needs of individuals and families affected by RYR-1-RD. Together, with your help, we have built a vibrant community of 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 solely dedicated to RYR-1-RD. With your participation and support, we continue to be a vital resource for the RYR-1-RD community.

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

How You Can Engage With Us in 2025:

• Join us for The RYR-1 Foundation International Family Conference 2025 Register by December 31, 2024, to receive a $100 discount per person

• Attend one of our webinars – these are free and easily accessible

Watch your email and follow us on social media for information and announcements about upcoming webinars

• Participate in our 2025 Advocacy Campaign

Help us advocate for an increase in federal funding for RYR-1-RD in the coming months

• Make sure to read our monthly newsletter, visit our website, and follow us on social media for the latest updates, event information, and patient resources 

Additional Plans for 2025 Include:

• Awarding $500,000 in new research grants

• Hosting the 2nd RYR-1-RD Patient-Led International Research Workshop

• Continuing to build collaborative relationships with other organizations such as the Muscular Dystrophy Association (MDA) 
• Participating in Research Conferences such as the Breakthroughs in Muscular Dystrophy Conference, which we attended in 2024

And we are just getting started! As we look to the New Year and the next decade, we know this is just the beginning of what we hope to achieve together with you and the entire RYR-1-RD community. Your participation will help us reach our ultimate goal of finding 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 join us in shaping the future. To reach these goals we also ask you to consider making a year-end donation to The RYR-1 Foundation by December 31, 2024. Your gift, no matter the amount, can make an impact in the RYR-1-RD community by expanding resources for affected individuals and families, supporting the International Family Conference, contributing to vital research, driving advocacy, and building awareness.

In addition to making an immediate difference, your year-end gift may also provide a potential tax benefit.

We are grateful that you are part of our community. With your ongoing participation and sustained support, we will accelerate our progress towards making our motto “Strength in Numbers,” a reality.

To donate you can go to our website. If you would like to donate through a will, trust, securities, or other means, please contact Executive Director Lindsay Goldberg. If you have any questions please contact Lindsay or Mike.

Once again, on behalf of the RYR-1-RD community and The RYR-1 Foundation, we sincerely thank you for your continued support.  

We wish you and your family Happy Holidays and New Year!

Best Wishes,

Lindsay Goldberg, BSN, RN
Executive Director, Patient Liaison & Co-Founder
The RYR-1 Foundation

Michael F. Goldberg, MD, MPH
Board President & Co-Chair of Research
The RYR-1 Foundation

Make Your Year-End Donation Now!