NATIONAL TUBEROUS SCLEROSIS ASSOCIATION
8737 COLESVILLE ROAD NO 400, SILVER SPRING, MD 20910 www.tsalliance.org

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Organizations Filed Purposes: THE NATIONAL TUBEROUS SCLEROSIS ASSOCIATION, D/B/A TSC ALLIANCE, IS DEDICATED TO FINDING A CURE FOR TUBEROUS SCLEROSIS COMPLEX WHILE IMPROVING THE LIVES OF THOSE AFFECTED.

FINDING A CURE FOR TUBEROUS SCLEROSIS COMPLEX WHILE IMPROVING THE LIVES OF THOSE AFFECTED.

RESEARCH PROGRAM STIMULATES AND SUPPORTS BASIC, TRANSLATIONAL, AND CLINICAL RESEARCH ON THE VARIOUS MANIFESTATIONS OF TUBEROUS SCLEROSIS COMPLEX (TSC) TO FURTHER THE DEVELOPMENT OF CLINICAL THERAPIES AND, ULTIMATELY, A CURE FOR TSC. DIRECTED BY STEVEN L. ROBERDS, PHD, CHIEF SCIENTIFIC OFFICER, THE TSC ALLIANCE RESEARCH PROGRAM BUILDS AND FOSTERS COLLABORATIONS BETWEEN BASIC AND CLINICAL RESEARCHERS BY COLLECTING AND DISTRIBUTING TSC NATURAL HISTORY DATA AND BIOSAMPLES, THROUGH COLLABORATIVE PRECLINICAL AND CLINICAL RESEARCH PROGRAMS, AND BY HOSTING BIENNIAL INTERNATIONAL TSC RESEARCH CONFERENCES.SINCE 1984, THE TSC ALLIANCE HAS INVESTED $27.7 MILLION INTO TSC RESEARCH PROJECTS THROUGH GRANTS AND CONTRACTS: $18.5 MILLION IN RESEARCH GRANTS AND POSTDOCTORAL FELLOWSHIPS, $4.4 MILLION INTO THE NATURAL HISTORY DATABASE AND BIOSAMPLE REPOSITORY, $4.1 MILLION INTO THE PRECLINICAL CONSORTIUM AND $0.7 MILLION INTO THE CLINICAL RESEARCH CONSORTIUM. GRANT AND FELLOWSHIP APPLICATIONS ARE REVIEWED IN A THREE-STEP PROCESS: (1) ALL APPLICATIONS ARE REVIEWED BY A COMMITTEE COMPRISED OF SCIENTISTS KNOWLEDGEABLE ABOUT THE TOPIC AREA FOR SCIENTIFIC MERIT AND OF CAREGIVERS OR ADULTS AFFECTED BY TSC FOR POTENTIAL IMPACT ON THE LIVES OF THOSE AFFECTED BY TSC; (2) THE SCIENCE AND MEDICAL COMMITTEE OF THE BOARD OF DIRECTORS EVALUATES THE GRANT REVIEW COMMITTEE'S RECOMMENDATIONS AND THE RELEVANCE OF THE APPLICATIONS TO THE TSC ALLIANCE'S FUNDING PRIORITIES; AND (3) THE BOARD OF DIRECTORS THEN REVIEWS THE RECOMMENDATIONS OF THE SCIENCE AND MEDICAL COMMITTEE AND MAKES FINAL APPROVAL FOR FUNDING. FOR A COMPLETE LIST OF CURRENTLY FUNDED PROJECTS AND AN ARCHIVE OF PAST AWARDEES, PLEASE VISIT TSALLIANCE.ORG/GRANTS.IMPLEMENTED IN 2006, THE TSC NATURAL HISTORY DATABASE CAPTURES CLINICAL DATA TO DOCUMENT THE IMPACT OF THE DISEASE ON A PERSON'S HEALTH OVER THEIR LIFETIME. THE DATABASE SERVES AS A RESOURCE OF INFORMATION THAT HELPS TSC RESEARCHERS BETTER UNDERSTAND THE PROGRESSION OF THE DISEASE, DESIGN HYPOTHESIS-DRIVEN QUESTIONS TO HASTEN THE DISCOVERY OF NEW TREATMENTS AND IDENTIFY PERSONS WITH TSC WHO ARE ELIGIBLE TO PARTICIPATE IN RESEARCH STUDIES. AS OF DECEMBER 2020, 2,268 PEOPLE WITH TSC WERE ENROLLED IN THE PROJECT FROM AMONG 18 U.S.-BASED SITES AND THROUGH THE TSC ALLIANCE. THE TSC ALLIANCE PROVIDES FUNDING TO PARTICIPATING CLINICS TO PERFORM DATA ENTRY, MONITORS THE INTEGRITY OF THE DATABASE, AND MAKES DATA AVAILABLE TO INVESTIGATORS TO ANSWER SPECIFIC RESEARCH QUESTIONS AND IDENTIFY POTENTIAL PARTICIPANTS FOR CLINICAL TRIALS AND STUDIES. IN 2020, THE TSC ALLIANCE ADDED A COVID-19 SUB-PROJECT TO ADDRESS DETAILED QUESTIONS ABOUT COVID-19 INFECTION IN INDIVIDUALS WITH TSC.BUILDING UPON THE NATURAL HISTORY DATABASE, THE TSC BIOSAMPLE REPOSITORY IS A TSC ALLIANCE-DIRECTED PROJECT INITIATED IN 2014 THAT WILL IMPACT RESEARCH OVER THE NEXT TEN YEARS OR MORE. THE TSC ALLIANCE'S SCIENCE AND MEDICAL COMMITTEE IDENTIFIED THIS AS A GAP THAT CAN ONLY BE FILLED EFFECTIVELY WITH LEADERSHIP OF THE TSC ALLIANCE, GUIDED BY A STEERING COMMITTEE OF CLINICIANS AND RESEARCHERS. HIGH-QUALITY BIOSAMPLES SUCH AS BLOOD, DNA, AND TISSUES LINKED TO DETAILED CLINICAL DATA ARE CRITICAL FOR RESEARCHERS TO UNDERSTAND WHY TSC IS SO DIFFERENT FROM PERSON TO PERSON. SAMPLES IN THE REPOSITORY ARE LINKED TO DETAILED CLINICAL DATA IN OUR EXISTING TSC NATURAL HISTORY DATABASE (NHD) AND ARE AVAILABLE TO QUALIFIED RESEARCHERS WORLDWIDE. SAMPLES ARE HOUSED AT AND DISTRIBUTED FROM THE VAN ANDEL INSTITUTE IN GRAND RAPIDS, MI, UNDER CONTROL OF THE TSC ALLIANCE. THE TSC BIOSAMPLE REPOSITORY ENDED 2020 WITH 1,571 BIOSAMPLES: 421 BLOOD SAMPLES FROM INDIVIDUALS WITH TSC ENROLLED IN THE NHD, 358 BUCCAL SAMPLES FROM INDIVIDUALS WITH TSC ENROLLED IN THE NHD, 29 TISSUE SAMPLES FROM INDIVIDUALS WITH TSC ENROLLED IN THE NHD, 374 BLOOD SAMPLES FROM THE TSC CLINICAL RESEARCH CONSORTIUM COLLABORATIVE PROJECTS, AND 389 DNA SAMPLES FROM THE TSC AUTISM CENTER OF EXCELLENCE NETWORK AUTISM BIOMARKER STUDY. WE CONTINUE TO GROW THE BIOSAMPLE REPOSITORY BY COLLECTING BLOOD SAMPLES ANNUALLY FROM PARTICIPANTS WHO VOLUNTEER TO DO SO, ENABLING RESEARCHERS TO STUDY CHANGES IN PROTEINS AND OTHER MOLECULES IN THE BLOOD OVER TIME; ADDING ADDITIONAL TSC CLINICS AS PARTICIPATING BIOSAMPLE REPOSITORY COLLECTION SITES; AND COLLECTING BLOOD USING MOBILE PHLEBOTOMY, ENABLING PEOPLE TO DONATE SAMPLES REGARDLESS OF WHERE THEY LIVE IN THE US AND WHETHER THEY ARE SEEN AT A TSC CLINIC. IN 2020, WE ACQUIRED 65 BLOOD SAMPLES VIA MOBILE PHLEBOTOMY, INCLUDING 4 FROM PARTICIPANTS WHO WERE DIAGNOSED WITH COVID-19. TO ENCOURAGE AND ENABLE MORE RESEARCHERS TO UTILIZE BIOSAMPLES, THE TSC ALLIANCE FUNDED THREE SEED GRANTS IN 2020, TOTALING $60,000, TO LABS WHO SUBMITTED MERITORIOUS AND INNOVATIVE IDEAS FOR RESEARCH ON TSC BIOSAMPLES.THE TSC ALLIANCE LAUNCHED THE TSC PRECLINICAL CONSORTIUM IN 2015 TO HELP ADVANCE MORE DRUG CANDIDATES INTO CLINICAL TESTING. TO ACHIEVE THIS END, THE PRECLINICAL CONSORTIUM PROVIDES THE INFRASTRUCTURE TO FOSTER COLLABORATION BETWEEN ACADEMIA AND PHARMACEUTICAL INDUSTRY RESEARCHERS AND FOR ACCESS TO RESOURCES DESIGNED TO HELP FACILITATE DRUG DEVELOPMENT IN TSC. COLLABORATING WITH THE TSC COMMUNITY, THE CONSORTIUM HAS IDENTIFIED AND IMPLEMENTED ROBUST AND REPRODUCIBLE CELL AND ANIMAL MODELS FOR TSC MANIFESTATIONS INCLUDING TUMORS, EPILEPSY, AND TSC-ASSOCIATED NEUROPSYCHIATRIC DISORDERS (TAND). THE TSC ALLIANCE HAS LICENSES TO USE SPECIFIC TSC MOUSE MODELS FOR EXPERIMENTS CARRIED OUT BY THE PRECLINICAL CONSORTIUM, AND ALL MOUSE LICENSE AGREEMENTS INCLUDE THE RIGHTS FOR THE TSC ALLIANCE TO PERFORM EXPERIMENTS UNDER CONTRACT FOR COMMERCIAL ENTITIES. THIS ENSURES DATA GENERATED BY THE PRECLINICAL CONSORTIUM CAN BE USED TO ACCELERATE THE DEVELOPMENT OF NEW TREATMENTS BY COMMERCIAL ENTITIES AS WELL AS ACADEMIC INVESTIGATORS. EXPERIMENTS ARE EXECUTED AT PARTNERING RESEARCH INSTITUTIONS TO ENSURE CONSISTENCY IN TESTING, DATA ACQUISITION AND INTERPRETATION. EPILEPSY STUDIES ARE CONDUCTED AT PSYCHOGENICS (US), AND THE TUMOR GRAFT MODEL AND CELL-BASED ASSAYS ARE CONDUCTED AT PORSOLT (FRANCE). THE VAN ANDEL RESEARCH INSTITUTE, A NON-PROFIT RESEARCH ORGANIZATION, MAINTAINS A COLONY OF TSC2+/- AJ MICE AND L7-CRE-TSC2 MICE ON BEHALF OF THE PRECLINICAL CONSORTIUM. TWICE A YEAR, THE PRECLINICAL CONSORTIUM HAS A CALL FOR COMPOUND NOMINATIONS TO CONTINUE TO REFRESH OUR PIPELINE BASED ON THE LATEST DATA AND NOVEL IDEAS. THE NOMINATIONS ARE PEER-REVIEWED BY CONSORTIUM MEMBERS AND PRIORITIZED BY THE PRECLINICAL CONSORTIUM STEERING COMMITTEE FOR FUNDING BY THE TSC ALLIANCE. IF THE TSC ALLIANCE FUNDS THE STUDY, THE DATA ARE SHARED WITH THE CONSORTIUM FOR TRANSPARENCY AND STIMULATION OF ADDITIONAL HYPOTHESES. AN ANNUAL FACE-TO-FACE MEMBERS MEETING IS TYPICALLY HELD EACH YEAR IN APRIL TO KEEP MEMBERS ENGAGED AND ALIGNED ON THE CONSORTIUM'S STRATEGY AND PRIORITIES. PARTNERSHIP WITH PHARMACEUTICAL COMPANIES IS AN IMPORTANT ASPECT OF THE CONSORTIUM, AS THESE ORGANIZATIONS HAVE THE INFRASTRUCTURE TO EFFICIENTLY MOVE PRECLINICAL RESEARCH FINDINGS TO CLINICAL TESTING AND EVENTUALLY COMMERCIAL DISTRIBUTION. AS OF DECEMBER 2020, 7 COMPANIES WERE ACTIVE MEMBERS OF THE CONSORTIUM. THE CONSORTIUM HAS HAD 14 COMPANY MEMBERS SINCE 2016. THERE IS NATURAL TURNOVER OF INDUSTRY MEMBERSHIP, PRINCIPALLY DRIVEN BY RESEARCH DATA GENERATED BY TESTING THEIR DRUGS. DUE TO INTELLECTUAL PROPERTY CONSIDERATIONS, MOST COMPANIES FULLY FUND THEIR STUDIES THROUGH THE TSC ALLIANCE, WHICH ALLOWS THE COMPANY TO KEEP ITS DATA CONFIDENTIAL. THESE CONFIDENTIAL STUDIES ALSO INCUR A 10% OVERHEAD PAID BY THE COMPANY, WHICH IS A SOURCE OF REVENUE FOR THE TSC ALLIANCE. SINCE INCEPTION, THE CONSORTIUM HAS HELPED ADVANCE THE EVALUATION OF DRUGS FOR TSC BY CONDUCTING 54 STUDIES, EVALUATING 44 CANDIDATE DRUGS. IN 2020, THE TSC PRECLINICAL CONSORTIUM EVALUATED 13 CANDIDATE DRUGS IN MOUSE MODELS AND SCREENED AN ADDITIONAL 10 COMPOUNDS IN CELL-BASED MODELS OF TSC.

SUPPORT SERVICES DEVELOPS PROGRAMS AND SERVICES THAT PROVIDE INDIVIDUALS WITH TSC DIRECT ACCESS TO INFORMATION, RESOURCES, AND SPECIALISTS EXPERIENCED IN THE DIAGNOSIS, TREATMENT AND MANAGEMENT OF TSC. IN 2020 THE TSC ALLIANCE FACILITATED 28,447 PEER-TO-PEER SUPPORTS FROM ADULT REGIONAL COORDINATORS (12 VOLUNTEERS IN 8 REGIONS NATIONWIDE), CLINIC AMBASSADORS (22 VOLUNTEERS IN 26 TSC CLINICS AND TSC CENTERS OF EXCELLENCE NATIONWIDE), DEPENDENT ADULT TRANSITION RESOURCE COORDINATORS (32 VOLUNTEERS IN 26 STATES AND PUERTO RICO), EDUCATION PARENT MENTORS (31 VOLUNTEERS IN 22 STATES), AND COMMUNITY ALLIANCE LEADERS (93 VOLUNTEERS IN 33 REGIONS ACROSS THE UNITED STATES) WITH ACTIVE FUTURE LEADERS (6 VOLUNTEERS) AND PAST FUTURE LEADERS (8 VOLUNTEERS) WHO HAVE AGREED TO REMAIN AS LEADERS FOR OTHER YOUNG ADULTS WITH TSC.EDUCATION PARENT MENTORS ATTENDED 115 SCHOOL MEETINGS (IEPS, EVALUATION TEAM MEETINGS, 504 PLAN MEETINGS, RESOLUTION MEETINGS, AND MEDIATIONS) IN PERSON, THROUGH SKYPE/ FACETIME, AND VIA CONFERENCE CALLS TO SUPPORT FAMILIES IN ATTAINING EDUCATIONAL SERVICES FOR THEIR CHILDREN THROUGHOUT THE COUNTRY. SEVERAL SCHOOL SYSTEMS ALSO REQUESTED "TSC 101" IN AN EFFORT TO HELP THEM UNDERSTAND THE COMPLEXITIES OF TSC AND LEARNING ISSUES FOR CHILDREN WITH TSC. THE COMMUNITY PROGRAMS TEAM SUPPORTS A NETWORK OF 33 VOLUNTEER BRANCHES OF THE ORGANIZATION, CALLED COMMUNITY ALLIANCES, DIVIDED INTO 2 REGIONS, WITH A COMMUNITY PROGRAM MANAGER ASSIGNED TO EACH REGION TO MANAGE THE DAY-TO-DAY ACTIVITIES WITHIN THEIR REGIONS. AS OF DECEMBER 31, 2020, THERE WERE 98 LEADERSHIP POSITIONS (50 COMMUNITY ALLIANCE CHAIRS, CO-CHAIRS, VICE CHAIRS AND 48 WALK/FUNDRAISING CHAIRS AND CO-CHAIRS) WITH 3 OPEN POSITIONS. IN 2020, THESE COMMUNITY ALLIANCES PROVIDED LOCAL TSC EDUCATION AND PERSONAL SUPPORT THROUGHOUT THE COUNTRY TO 4,988 FAMILIES/INDIVIDUALS LIVING WITH TSC. TO HELP THE TSC COMMUNITY NAVIGATE THE CHALLENGES OF COVID-19, THE TSC ALLIANCE CREATED A SECTION OF OUR WEBSITE DEDICATED TO SHARING IMPORTANT INFORMATION ABOUT THE PANDEMIC SPECIFICALLY CURATED TO MEET THE NEEDS OF THOSE IMPACTED BY TSC. THE TSC ALLIANCE COVID-19 LANDING PAGE, WWW.TSALLIANCE.ORG/COVID-19, INCLUDES RESOURCES FOR TSC MEDICAL PROFESSIONALS (WITH THE HELP OF OUR PROFESSIONAL ADVISORY BOARD); VACCINE POSITION STATEMENT; LISTING OF TSC CLINICS OFFERING TELEHEALTH; FDA DRUG SHORTAGES; SUPPLY CHAIN UPDATES; CO-PAY SAVINGS PROGRAMS DURING COVID-19; COVID-19 FAQS; COMMUNITY EDUCATIONAL RESOURCES; AND WEBINAR SERIES. THERE WERE 23,447 VIEWS ON THE COVID-19 LANDING PAGE IN 2020.THE TSC ALLIANCE ALSO LAUNCHED A WEEKLY EDUCATIONAL SERIES ON MARCH 23 WITH PROGRAMMING LASTING 11 WEEKS. THIS EFFORT INCLUDED FIVE VIRTUAL TOWN HALLS IN PARTNERSHIP WITH OTHER NONPROFIT ORGANIZATIONS AS WELL AS HOSTING TWO WORLD FORUM WEBINARS FOR TSCI ON MAY 12 AND 26. - MARCH 20: TSC/LAM VIRTUAL TOWN HALL: COVID-19 UPDATE, CO-HOSTED WITH THE LAM FOUNDATION- APRIL 10: VIRTUAL TOWN HALL: ACCESSIBILITY TO CARE, CO-HOSTED WITH THE CHILD NEUROLOGY FOUNDATION AND DUP15Q ALLIANCE- MAY 1: VIRTUAL TOWN HALL: ACCESSIBILITY TO TREATMENTS (INCLUDING DRUG SUPPLY), CO-HOSTED WITH THE DRAVET SYNDROME FOUNDATION AND LGS FOUNDATION- MAY 15: VIRTUAL TOWN HALL: RESCUE MEDS, SEIZURE TRACKER AND A FUTURE GLIMPSE AT FORECASTING SEIZURE RISK, CO-HOSTED WITH SEIZURE TRACKER- MAY 22: TSC/LAM VIRTUAL TOWN HALL: MOVING ON FROM SHELTER IN PLACE, CO-HOSTED WITH THE LAM FOUNDATION THE TSC ALLIANCE ALSO PRESENTED 15 WEBINARS ON TOPICS OF RELEVANCE TO TSC INDIVIDUALS AND FAMILIES, INCLUDING SCHOOLING AT HOME, ANXIETY, SELF-CARE, FILING FOR UNEMPLOYMENT, RESEARCH UPDATES, COMMUNITY PROGRAMS UPDATE, REMOTE BEHAVIORAL INTERVENTIONS AND OTHERS. NINE OPEN FORUMS WERE HELD, PROVIDING A PLATFORM FOR CAREGIVERS, TEENS, SIBLINGS AND ADULTS WITH TSC TO OPENLY DISCUSS ISSUES RELATED TO COVID-19 AND TSC. IN TOTAL, THERE WERE 9,151 VIEWS OF THE WEBINAR AND TOWN HALL SERIES. THE TSC ALLIANCE CO-HOSTED 3 VIRTUAL REGIONAL CONFERENCES WITH THE LAM FOUNDATION FEATURING REGIONAL PROFESSIONALS IN MEMPHIS, GAINSVILLE AND DENVER. THESE EVENTS ATTRACTED A TOTAL OF 818 REGISTERED ATTENDEES, INCLUDING 636 ATTENDEES ON THE DAY OF THE CONFERENCE, 1,106 SUBSEQUENT VIDEO VIEWS, AND 424 VISITS TO THE VIRTUAL EXHIBIT HALLS. THERE WERE 21 SPEAKERS FROM 12 DIFFERENT INSTITUTES, REPRESENTING TSC CLINICS, TSC CENTERS OF EXCELLENCE OR LAM CLINICS.

PUBLIC HEALTH EDUCATION HEIGHTENS AWARENESS OF TSC THROUGHOUT THE GENERAL PUBLIC TO BROADEN THE SCOPE OF SUPPORT AND UNDERSTANDING BEYOND TSC INDIVIDUALS AND THEIR FAMILIES. DURING 2020, THE TSC ALLIANCE PRODUCED TWO ISSUES OF ITS NATIONAL MAGAZINE, PERSPECTIVE, WHICH IS MAILED TO APPROXIMATELY 14,500 CONSTITUENTS AS WELL AS POSTED ON THE WEBSITE. THE TSC ALLIANCE'S WEBSITE INCREASES AWARENESS AND PROVIDES EXTENSIVE EDUCATION THROUGH AN AVERAGE OF MORE THAN 22,300 UNIQUE VISITORS EACH MONTH. THE TSC ALLIANCE ALSO RELIES HEAVILY ON SOCIAL MEDIA TO EDUCATE CONSTITUENTS AND PROMOTE NEW RESOURCES AND EVENTS. ITS FACEBOOK GROUP BOASTS MORE THAN 10,000 MEMBERS, WHILE ITS TWITTER AND INSTAGRAM ACCOUNTS BOTH HAVE 2,400-PLUS FOLLOWERS. THE TSC ALLIANCE'S PODCAST SERIES, CALLED TSC NOW, PRODUCED 11 EPISODES WITH 1,906 DOWNLOADS. SIX ISSUES OF THE ELECTRONIC, TSC MATTERS, WERE DISTRIBUTED TO 1,006 SUBSCRIBERS. THE TSC ALLIANCE'S ONLINE EDUCATIONAL PLATFORM, TSC ACADEMY, INTRODUCED 3 NEW COURSES COVERING THE TOPICS OF CBD, FDA REVIEW PROCESS AND STATE AND LOCAL ADVOCACY.TO INCREASE PUBLIC AWARENESS, THE TSC ALLIANCE PARTICIPATED IN THE SIXTH ANNUAL TSC GLOBAL AWARENESS DAY ON MAY 15 AS WELL AS TSC AWARENESS MONTH THROUGHOUT MAY. THE TSC ALLIANCE AGAIN HEAVILY PROMOTED INFANTILE SPASMS AWARENESS WEEK, DECEMBER 1 TO 7. THIS AWARENESS CAMPAIGN INCLUDED A SOCIAL MEDIA CAMPAIGN AND TARGETED NATIONAL PRINT PUBLICATIONS, MEDICAL TRADE MAGAZINES AND PODCASTS, RESULTING IN 10 MEDIA PLACEMENTS, 31.7 MILLION-PLUS IMPRESSIONS AND 550-PLUS SOCIAL ENGAGEMENTS. IN 2020, THE TSC ALLIANCE'S VARIOUS NEWS RELEASES CULMINATED IN 694 MILLION IMPRESSIONS.

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