RYR-1 FOUNDATION
PO BOX 13312, PITTSBURGH, PA 15243 www.ryr1.org

---

Organizations Filed Purposes: THE MISSION OF THE RYR-1 FOUNDATION IS TO SUPPORT RESEARCH LEADING TO AN EFFECTIVE TREATMENT OR A CURE FOR RYR-1 RELATED DISEASES THROUGH SUPPORTING RESEARCH, PHYSICIAN EDUCATION, AND PATIENT & FAMILY SUPPORT AND ADVOCACY.

PATIENT OUTREACH AND EDUCATION - THE FOUNDATION RAISES AWARENESS THROUGH RESOURCES ON ITS WEBSITE, INCLUDING THE LATEST MEDICAL LITERATURE, AS WELL AS DIRECT MEETINGS WITH PHYSICIANS AROUND THE WORLD. THE FOUNDATION SERVES AS A RESOURCE FOR PATIENTS AND THEIR FAMILIES THROUGH ITS WEBSITE, OTHER FORMS OF SOCIAL MEDIA, AND FAMILY CONFERENCES.

RESEARCH GRANTS - THE FOUNDATION MAKES GRANTS TO RESEARCHERS INTERESTED IN RYR-1 MYOPATHY. AS THE ONLY ORGANIZATION SOLELY DEDICATED TO RYR-1 MYOPATHY, WE HOPE TO BE ABLE TO PROMOTE RESEARCH IN THIS AREA.

SCIENTIFIC MEETINGS - RYR-1-RELATED MYOPATHY (RYR-1-RM) IS A RARE, INHERITED, CONGENITAL MYOPATHY THAT AFFECTS APPROXIMATELY 1/90,000 INDIVIDUALS. LIKE MANY ORPHAN DISEASES," THERE ARE A LIMITED NUMBER OF RESEARCHERS STUDYING THIS CONDITION. FURTHER, THERE ARE NO REGULARLY OCCURRING MEETINGS THAT SPECIFICALLY ADDRESS RYR-1-RM. SCIENTIFIC MEETINGS FOCUSED ON AN ORPHAN DISEASE, SUCH AS RYR-1-RM, SERVE SEVERAL PURPOSES: 1) TO DISSEMINATE NEW KNOWLEDGE TO LEADERS IN THE FIELD OF RYR-1-RM; 2) TO PROVIDE A FORUM FOR FUNDED RESEARCHERS TO RECEIVE CRITICAL REVIEWS AND SUGGESTIONS FOR IMPROVEMENTS; 3) TO ALLOW THE RYR-1 FOUNDATION TO ASSESS THE QUALITY OF THE RESEARCH IN A "GIVE AND TAKE" ENVIRONMENT; AND 4) TO ENCOURAGE MEDICAL/RESEARCH TRAINEES TO PURSUE A CAREER IN RYR-1-RM. THE RYR-1 FOUNDATION HAS HOSTED SEVEN, ONE-DAY SCIENTIFIC MEETINGS OVER THE PAST FOUR YEARS. PARTICIPANTS IN THESE MEETINGS INCLUDE MEMBERS OF THE RYR-1 FOUNDATION'S SCIENTIFIC ADVISORY BOARD (SAB), RESEARCHERS RECEIVING FUNDING FROM THE RYR-1 FOUNDATION, AND REPRESENTATIVES FROM THE CLINICAL, RESEARCH, AND PATIENT ADVOCACY COMMUNITIES. THE FOCUS OF THESE MEETINGS IS TO DISSEMINATE RESEARCH FINDINGS FROM RECIPIENTS OF RYR-1 FOUNDATION GRANTS. IN THIS SETTING, THE RESEARCHERS RECEIVE FEEDBACK FROM THE SAB; THIS DETERMINES IF THE RESEARCHERS HAVE MET THEIR MILESTONES IN ORDER TO RECEIVE ADDITIONAL FUNDING. IN ADDITION TO FUNDED RESEARCHERS, NON-FUNDED INVESTIGATORS WHO HAVE IMPORTANT INSIGHTS ARE INVITED TO SPEAK. TIME IS ALSO DEVOTED TO STRATEGIC PLANNING FOR FUTURE RESEARCH PRIORITIES AND FUNDING. PERHAPS MOST IMPORTANTLY, THESE MEETINGS PROVIDE A FORUM FOR RESEARCHERS FROM AROUND THE WORLD TO SHARE IDEAS AND DEVELOP PLANS FOR FUTURE COLLABORATIONS.

---

---

Data for this page was sourced from XML published by IRS (public 990 form dataset) from: https://s3.amazonaws.com/irs-form-990/202032549349300503_public.xml