Friedreichs Ataxia Research Alliance Fara

TO TREAT AND CURE FRIEDREICH'S ATAXIA (FA) BY ADVANCING RESEARCH, AWARENESS AND PARTNERSHIPS.

SEE SCHEDULE O

WORKSHOPS, SYMPOSIA, & CONFERENCESEDUCATION: FARA HOSTED 5 RESEARCH RECEPTIONS AND SYMPOSIUMS TO SHARE INFORMATION ABOUT THE FA RESEARCH PIPELINE, CURRENT CLINICAL TRIALS, CLINICAL MANAGEMENT OF FA, MENTAL WELLNESS, AND MORE. OVER 500 COMMUNITY MEMBERS ATTENDED THESE EVENTS TO LEARN AND CONNECT WITH EACH OTHER. VIRTUAL EDUCATION SESSIONS, INCLUDING FLASH TALKS, WEBINARS, AND 2 MINUTE MECHANISM VIDEOS, WERE ALSO SHARED THROUGHOUT THE YEAR.

CLINICAL RESEARCH INFRASTRUCTUREIN ADDITION TO RESEARCH GRANTS, FARA FUNDS THE ONGOING DEVELOPMENT OF DOMAIN RESOURCES IN THE FORM OF VITAL CLINICAL RESEARCH INFRASTRUCTURE. CLINICAL RESEARCH INFRASTRUCTURE REFERS TO THE RESOURCES NEEDED TO FACILITATE ANY TYPE OF RESEARCH, INCLUDING CLINICAL TRIALS THAT INVOLVE PATIENTS. THESE RESOURCES INCLUDE PROGRAMS LIKE: FRIEDREICH'S ATAXIA GLOBAL PATIENT REGISTRY: THE FRIEDREICH'S ATAXIA GLOBAL PATIENT REGISTRY (FAGPR) IS THE ONLY WORLDWIDE REGISTRY OF FRIEDREICH'S ATAXIA PATIENTS. THE GOALS OF THE FAGPR ARE TO COLLECT INFORMATION ON ALL FA PATIENTS IN ONE REGISTRY, TO DEVELOP THE REGISTRY INTO A POWERFUL RESOURCE FOR RESEARCH, AND TO ENGAGE THE FA COMMUNITY IN STUDIES AIMED AT ADVANCING OUR KNOWLEDGE OF FA AND THE TREATMENTS BEING DEVELOPED. FARA PARTNERS WITH INTERNATIONAL PATIENT ADVOCACY ORGANIZATIONS THROUGH A GOVERNANCE BOARD TO ENSURE MULTI-STAKEHOLDER ENGAGEMENT AND OVERSIGHT OF THE FAGPR. MORE THAN 1000 INDIVIDUALS WITH FA ARE ENROLLED AND FAGPR WAS USED TO RECRUIT FOR SEVERAL CLINICAL TRIALS AND MANY CLINICAL RESEARCH STUDIES. TO LEARN MORE, VISIT CUREFA.NET/REGISTRY. FA GLOBAL CLINICAL CONSORTIUM: FARA AND THE FA COMMUNITY'S DEDICATION TO COLLECTING NATURAL HISTORY STUDY DATA OVER THE PAST TWO DECADES PROVED INSTRUMENTAL IN THE APPROVAL OF THE FIRST-EVER TREATMENT FOR FA. THROUGH THE FA GLOBAL CLINICAL CONSORTIUM (FA GCC), FARA HAS REDOUBLED ITS INVESTMENT IN NATURAL HISTORY DATA BY ENABLING THE TRANSITION TO AN INDUSTRY-LEADING DATA COLLECTION PLATFORM AND A UNIFIED GLOBAL NATURAL HISTORY PROTOCOL (UNIFAI) RESULTING FROM THE HARMONIZATION OF TWO WELL-ESTABLISHED NATURAL HISTORY STUDIES, THE FA CLINICAL OUTCOME MEASURES STUDY (FA-COMS) AND THE EUROPEAN FRIEDREICH'S ATAXIA CONSORTIUM FOR TRANSLATIONAL STUDIES (EFACTS). THE FA GCC AND THE UNIFAI STUDY HAVE BEEN EXPANDED TO MAKE PATIENT CONTRIBUTIONS TO NATURAL HISTORY EVEN MORE POWERFUL IN UNDERSTANDING FA, EVALUATING THE IMPACT OF TREATMENT OPTIONS FOR FA, ACCELERATING THE DEVELOPMENT OF NEW THERAPIES AND IMPROVING OUTCOMES FOR THOSE LIVING WITH FA. AT EACH UNIFAI STUDY VISIT THERE ARE MORE THAN 1400 DATA ELEMENTS RECORDED. IN 2023, THE FA GCC HAD ACTIVE PARTICIPATION FROM 54 INVESTIGATORS FROM 33 SITES REPRESENTING 18 COUNTRIES ALONG WITH REPRESENTATIVES FROM PATIENT ADVOCACY AND RESEARCH ORGANIZATIONS. FA GCC RESEARCH ACTIVITIES: THE FA GCC LEADERSHIP IDENTIFIED INITIAL SCIENTIFIC PRIORITIES AND CREATED WORKGROUPS TO ADDRESS THESE PRIORITIES. THE FOLLOWING WORK GROUPS, EACH MADE UP OF 5-10 CONSORTIUM MEMBERS, MET REGULARLY TO ADDRESS GAPS IN CURRENT FA RESEARCH: CARDIAC NATURAL HISTORY, LATE-STAGE SYMPTOMS, PEDIATRIC / PRESYMPTOMATIC, BIO-SAMPLES, MOOD AND COGNITION, AND PATIENT ADVOCACY/ADVISORY TEAM. IMPACT AND FUTURE DIRECTION: THE CONSORTIUM HAS A LONG-TERM OBJECTIVE OF FOSTERING MULTILATERAL RESEARCH AND COLLABORATION ACROSS CONTINENTS, UNIFYING GLOBAL OPINIONS TO REGULATORS AND INDUSTRY PARTNERS, ACCELERATING THE DEVELOPMENT OF NEW THERAPIES, AND IMPROVING OUTCOMES FOR THOSE LIVING WITH FA. ADDITIONAL INFORMATION ABOUT FARA'S PROGRAMS IN 2023 CAN BE ACCESSED VIA THE ANNUAL REPORT AT: CUREFA.ORG

AWARENESS, EDUCATION, AND OUTREACH PROGRAMSAWARENESS: FRIEDREICH ATAXIA (FA) IS A RARE DISEASE; AFFECTING 1 IN 50,000 INDIVIDUALS. FARA IS DEDICATED TO ADVOCACY AND RAISING AWARENESS FOR FA. FARA HAS UTILIZED BOTH TRADITIONAL AND SOCIAL MEDIA STRATEGIES TO BRING GREATER AWARENESS TO FA IN THE GENERAL PUBLIC AND TO ENGAGE AND EDUCATE THE FA COMMUNITY. FOR EXAMPLE, FARA CONDUCTS AN ANNUAL A SOCIAL MEDIA CAMPAIGN THAT ENCOURAGES COMMUNITY PARTICIPATION LEADING UP TO FA AWARENESS DAY. ADVOCACY: FARA AND THE NATIONAL ATAXIA FOUNDATION (NAF) PARTNERED ON SEVERAL ADVOCACY INITIATIVES RELEVANT TO THE ATAXIA COMMUNITY. MOST NOTABLY, THE EFFORTS OF THE TWO ORGANIZATIONS RESULTED IN HEREDITARY ATAXIA BEING ADDED TO THE CONGRESSIONALLY DIRECTED MEDICAL RESEARCH PROGRAM (CDMRP) AT THE DEPARTMENT OF DEFENSE IN FISCAL YEAR (FY) 23, RESULTING IN SEVEN FA RESEARCHERS BEING RECOMMENDED FOR FUNDING TOTALING OVER $10 MILLION. FARA AND NAF WERE ALSO SUCCESSFUL IN GETTING A RESOLUTION PASSED DECLARING SEPTEMBER 25, 2023 NATIONAL ATAXIA AWARENESS DAY (S.RES 807) AND HOSTING THE FIFTH UNITED AGAINST ATAXIA HILL DAY- WITH 86 CONGRESSIONAL MEETINGS FEATURING THE PARTICIPATION OF 64 FARA AND NAF ADVOCATES FROM 33 STATES. ELEVATING THE FA VOICE: INDIVIDUALS AND FAMILIES LIVING WITH FA PROVIDE EXPERT INSIGHTS NECESSARY TO ADVANCE MEANINGFUL AND ACCESSIBLE TREATMENTS. THIS PAST YEAR, MEMBERS OF THE FA COMMUNITY ADVOCATED FOR FAIR COVERAGE OF SKYCLARYS AT 5 STATE MEDICAID MEETINGS. INDIVIDUALS WITH FA AND CAREGIVERS ALSO PROVIDED GUIDANCE FOR THE DESIGN OF A PATIENT PREFERENCE STUDY, WHICH WILL INVESTIGATE THE FA COMMUNITY'S ATTITUDES TOWARDS THE RISKS AND BENEFITS OF GENE THERAPY. ENGAGING WITH STAKEHOLDERS: THROUGH THE SHARING OF LIVED EXPERIENCES, MEMBERS OF THE FA COMMUNITY RAISED AWARENESS OF FA AND PROVIDED FEEDBACK TO DIFFERENT STAKEHOLDERS INCLUDING RESEARCHERS, PHARMACEUTICAL PARTNERS, AND GENETIC COUNSELING STUDENTS, AT 17 DIFFERENT EVENTS. SEVERAL PHARMACEUTICAL PARTNERS WORKING ON FA TREATMENTS COMMUNICATED DIRECTLY WITH THE FA COMMUNITY AT WEBINARS AND RESEARCH RECEPTIONS THROUGHOUT THE YEAR. CULTIVATING COMMUNITY: IN 2023 FARA STROVE TO SUPPORT THE FA COMMUNITY BY PROVIDING AVENUES FOR CONNECTION AND COLLABORATION. THE FARA AMBASSADOR PROGRAM, A GROUP OF ADULTS WITH FA WHO VOLUNTEER TO SUPPORT FARA'S MISSION, GREW TO 82 MEMBERS FROM COUNTRIES AROUND THE WORLD. AMBASSADORS NOW REPRESENT 12 COUNTRIES GLOBALLY. PLUS 32 STATES THROUGHOUT THE US.