2021-04-12T12:33:19-05:00 2020-12-31 420714325 RSM US LLP 2021 L STREET NW SUITE 400 WASHINGTON DC 20036 990 2020-01-01 953018799 NATIONAL TUBEROUS SCLEROSIS ASSOCIATION NATI 3015629890 8737 COLESVILLE ROAD NO 400 SILVER SPRING MD 20910 KARI L ROSBECK PRESIDENT & CEO 3015629890 2021-04-12 1 KARI ROSBECK XXX-XX-XXXX ELIZABETH W HELLER P00397829 2022932200 2021-04-12 2 AAL1 IAL1 2020 2022-09-23 18:48:47Z TSC ALLIANCE KARI L ROSBECK 8737 COLESVILLE ROAD NO 400 SILVER SPRING MD 20910 5266631 0 X WWW.TSALLIANCE.ORG X 1975 CA FINDING A CURE FOR TUBEROUS SCLEROSIS COMPLEX WHILE IMPROVING THE LIVES OF THOSE AFFECTED. 27 27 22 2284 0 0 5527543 4345011 461658 792685 27826 11770 699207 -21302 6716234 5128164 617106 685788 0 0 2223956 2382792 0 0 941587 2798273 2353610 5639335 5422190 1076899 -294026 10351316 11657873 594729 2195312 9756587 9462561 X 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. 0 0 2543079 680788 901832 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. 698125 5000 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. 502083 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. 149611 GOVERNMENT RELATIONS EFFORTS FOCUS ON INCREASING FEDERAL AND STATE APPROPRIATIONS FOR TSC RESEARCH, RAISING AWARENESS, AND COLLABORATING WITH GOVERNMENT PARTNERS TO DRIVE TSC RESEARCH FORWARD AND IMPROVE CLINICAL CARE AND TREATMENT OPTIONS FOR INDIVIDUALS WITH TSC. ON THE STATE LEVEL, THE TSC ALLIANCE ADVOCATED FOR STATE FUNDING FOR TSC CENTERS IN MISSOURI AND MARYLAND, RESULTING IN AT LEAST $500,000 IN STATE APPROPRIATIONS. THE ANNUAL TSC ALLIANCE MARCH ON CAPITOL HILL TO ADVOCATE FOR FEDERAL FUNDING FOR THE TUBEROUS SCLEROSIS COMPLEX RESEARCH PROGRAM (TSCRP) AT THE DEPARTMENT OF DEFENSE'S (DOD) CONGRESSIONALLY DIRECTED MEDICAL RESEARCH PROGRAM (CDMRP) TOOK PLACE IN EARLY MARCH 2020. MORE THAN 120 MEMBERS OF THE TSC COMMUNITY PARTICIPATED AND ASKED THEIR SENATORS AND REPRESENTATIVES TO SIGN ONTO DEAR COLLEAGUE LETTERS IN SUPPORT OF THE TSCRP. MANY OTHER ADVOCATES WHO WERE NOT ABLE TO TRAVEL TO WASHINGTON, DC MET WITH THEIR SENATORS AND REPRESENTATIVES IN THEIR HOME STATES TO MAKE THE CASE FOR CONTINUED FUNDING. THE FY21 HOUSE DEAR COLLEAGUE LETTER WAS CO-SPONSORED BY REPRESENTATIVES MARKWAYNE MULLIN (R-OK) AND DAVE LOEBSACK (D-IA) AND CLOSED WITH 216 SIGNERS. THE FY21 SENATE DEAR COLLEAGUE LETTER, SPONSORED BY SENATORS KEVIN CRAMER (R-ND) AND JON TESTER (D-MT), CLOSED WITH 35 SIGNATURES. SENATORS CRAMER AND TESTER WERE BOTH NEW SPONSORS FOR FY21. IN FY2021, THE TSC RESEARCH PROGRAM AT THE CDMRP RECEIVED AN $8 MILLION APPROPRIATION, BRINGING THE CUMULATIVE FUNDING TO $97 MILLION SINCE 2002 AS A RESULT OF OUR SUCCESSFUL GRASSROOTS EFFORTS. RESEARCH PERFORMED THROUGH THIS PROGRAM HAS RECENTLY LED TO ADDITIONAL CLINICAL TRIALS INCLUDING DETERMINING IF IMATINIB, A DRUG FDA-APPROVED FOR CANCER, CAN SAFELY IMPROVE LEVELS OF VEGF-D, A BIOMARKER OF LYMPHANGIOLEIOMYOMATOSIS (LAM), A LIFE-THREATENING LUNG MANIFESTATION OF TSC, FUNDED IN FY2013; TESTING A COMBINATION OF TWO DRUGS TO TREAT LAM FUNDED IN FY2012; A MULTI-SITE CLINICAL TRIAL TESTING THE EFFICACY OF AN EXPERIMENTAL TOPICAL RAPAMYCIN CREAM TO TREAT THE DISFIGURING FACIAL TUMORS, CALLED FACIAL ANGIOFIBROMAS, CAUSED BY TSC FUNDED IN FY2010; A CLINICAL RESEARCH NETWORK WAS CREATED TO TEST POTENTIAL NEW THERAPIES, TO VALIDATE BIOMARKERS, AND TO LEARN THE NATURAL HISTORY OF LEADING TO A CLINICAL TRIAL FUNDED IN FY2012. DATA OBTAINED FROM AN FY2010 TSCRP CLINICAL RESEARCH AWARD TO DEFINE EARLY AUTISM PREDICTORS IN TSC AND AN FY2014 TSCRP AWARD FOR A PILOT CLINICAL TRIAL IS BEING TESTED IN A LARGE, NIH-FUNDED CLINICAL TRIAL LOOKING AT THE EFFECTIVENESS OF A BEHAVIORAL INTERVENTION STRATEGY, JASPER, TO IMPROVE OUTCOMES IN CHILDREN WITH AUTISM. THE TSCRP HAS ALSO FUNDED RESEARCH TO DEVELOP ANIMAL MODELS OF TSC THAT HAVE SEIZURES, ENABLING A BETTER UNDERSTANDING OF THE ETIOLOGY OF TSC. BASED ON DATA FROM TSCRP-FUNDED ANIMAL MODELS OF TSC THAT HAVE SEIZURES AND SHARE PATHOLOGY RELATED TO THAT OF TRAUMATIC BRAIN INJURY, AN INDUSTRY-SPONSORED CLINICAL TRIAL DEMONSTRATED THE EFFECTIVENESS THE MTOR INHIBITOR, EVEROLIMUS, AT TREATING EPILEPSY IN MANY INDIVIDUALS WITH TSC. NONE OF THIS PROGRESS WOULD HAVE BEEN POSSIBLE WITHOUT THE CRITICAL SUPPORT PROVIDED THROUGH THE TSCRP. 66927 GLOBAL OUTREACH WORKS TO ADDRESS UNMET NEEDS WITHIN THE GLOBAL TSC COMMUNITY. THE PROGRAM PROVIDES THE OPPORTUNITY FOR THE TSC ALLIANCE TO SHARE EXPERIENCES AND ASSIST IN THE START-UP OF SUPPORT OF TSC-RELATED ORGANIZATIONS IN OTHER COUNTRIES. A GLOBAL ALLIANCE IS A STRUCTURED GROUP OF EMPOWERED AND CARING VOLUNTEERS WHO WORK CLOSELY WITH THE TSC ALLIANCE TO FACILITATE LOCAL CONNECTIONS FOR INDIVIDUALS AND FAMILIES AFFECTED BY TSC AND RAISE REVENUE AND AWARENESS WHILE SUPPORTING THE MISSION OF THE ORGANIZATION. THE TSC ALLIANCE HAS SIX GLOBAL PARTNERSHIPS, INCLUDING: TS ALLIANCE OF ISRAEL, TS CANADA ST, TS ALLIANCE OF MEXICO, TS ALLIANCE FOUNDATION (IN THAILAND), HUNGARIAN FOUNDATION FOR TUBEROUS SCLEROSIS, AND TS ALLIANCE OF INDIA. THIS TS ALLIANCE RECOGNIZES 10 TSC CLINICS IN GLOBAL ALLIANCE COUNTRIES. 24324 PROFESSIONAL EDUCATION EXPANDS PROGRAMS TO TARGET RESEARCHERS AND HEALTHCARE PROVIDERS CARING FOR INDIVIDUALS WITH TSC, MEDICAL STUDENTS, GENETIC COUNSELORS AND EDUCATORS TO MINIMIZE THE CONSEQUENCES OF IGNORANCE AND MISINFORMATION. THE TSC ALLIANCE PARTICIPATED IN AND PRESENTED AT 22 PROFESSIONAL MEETINGS IN 2020 INCLUDING: AMERICAN THORACIC SOCIETY (ATS) BOARD MEETING; NATIONAL INSTITUTE FOR NEUROLOGICAL DISORDERS AND STROKE (NINDS) COUNCIL MEETING; EPILEPSY LEARNING HEALTHCARE SYSTEM (ELHS) HIGH-LEVEL VIRTUAL LEARNING SESSION; DRUG INFORMATION ASSOCIATION (DIA) CONFERENCE ON PATIENT ENGAGEMENT; NINDS NONPROFIT FORUM; ATS PUBLIC ADVISORY ROUNDTABLE (ATS PAR) MEET THE EXPERTS, PATIENT/FAMILY EDUCATION FORUM; EPILEPSY LEADERSHIP COUNCIL (ELC) STEERING COMMITTEE MEETING; VIRTUAL WORLD ORPHAN DRUG CONGRESS; VIRTUAL EPILEPSY PIPELINE CONFERENCE; EVERYLIFE FOUNDATION FOR RARE DISEASES NEWBORN SCREENING BOOTCAMP; HEALTH RESEARCH ALLIANCE (HRA) MEMBERS MEETING; ATS LEADERSHIP SUMMIT; GLOBAL GENES RARE PATIENT ADVOCACY UN-SUMMIT; FASTERCURES PARTNERSHIP MATURITY MODEL WORKSHOP; ELHS FALL LEARNING SESSION; GLOBAL GENES: HOW PATIENTS IMPACT THE 505(B)(2) REGULATORY PROCESS; NATIONAL ASSOCIATION OF MANAGED CARE PHYSICIANS; INTERNATIONAL TSC RESEARCH CONFERENCE; EPILEPSY DAY AT DISNEYLAND; GLOBAL GENES RARE ENTREPRENEUR'S BOOTCAMP; AMERICAN EPILEPSY SOCIETY; AND MILKEN INSTITUTE FUTURE OF HEALTH SUMMIT. 240862 3984149 1 1 0 1 0 0 0 0 0 1 1 0 0 1 1 0 0 1 0 0 1 1 0 0 1 0 0 1 0 1 0 0 0 0 0 0 0 0 1 0 0 0 0 1 0 0 0 1 30 0 1 22 1 0 0 0 0 0 1 1 0 0 0 0 0 0 X 27 27 0 0 0 0 1 1 0 1 1 0 1 1 1 1 1 1 1 1 1 1 0 AL AR CA FL GA HI IL IN KS KY MD MA MI MN MS NH NJ NM NY NC OR PA RI SC TN UT VA WI WV X X X THE ORGANIZATION 3015629890 8737 COLESVILLE ROAD NO 400 SILVER SPRING MD 20910 CHRIS RUSSELL CHAIR 5.00 0.00 X X 0 0 0 PETER CRINO MD PHD VICE CHAIR 5.00 0.00 X X 0 0 0 TIM DILLS IMMEDIATE PAST CHAIR 5.00 0.00 X X 0 0 0 LAURA MARKS SECRETARY 5.00 0.00 X X 0 0 0 WILLIAM JOSEPH TREASURER 5.00 1.00 X X 0 0 0 JULIE BLUM BOARD MEMBER 2.00 0.00 X 0 0 0 MATT BOLGER BOARD MEMBER 2.00 0.00 X 0 0 0 CASSANDRA CARROLL BOARD MEMBER 2.00 0.00 X 0 0 0 MARK CARROLL BOARD MEMBER 2.00 1.00 X 0 0 0 SARA CHIEFFO BOARD MEMBER 2.00 0.00 X 0 0 0 DAVID COIT BOARD MEMBER 2.00 0.00 X 0 0 0 MARGARET COX BOARD MEMBER 2.00 0.00 X 0 0 0 BONNIE HOGUE DUFFY BOARD MEMBER 2.00 0.00 X 0 0 0 STEVEN GOLDSTEIN BOARD MEMBER 2.00 0.00 X 0 0 0 ROB GRANDIA BOARD MEMBER 2.00 0.00 X 0 0 0 DANA HOLINKA BOARD MEMBER 2.00 0.00 X 0 0 0 DARCY A KRUEGER BOARD MEMBER 2.00 0.00 X 0 0 0 HEATHER LENS BOARD MEMBER 2.00 0.00 X 0 0 0 JIM MAGINN BOARD MEMBER 2.00 0.00 X 0 0 0 DIANE MCSWAIN BOARD MEMBER 2.00 0.00 X 0 0 0 MUSTAFA SAHIN BOARD MEMBER 2.00 0.00 X 0 0 0 SEAN SHILLINGER BOARD MEMBER 2.00 0.00 X 0 0 0 JUDY SHOULAK BOARD MEMBER 2.00 0.00 X 0 0 0 MATTHEW SIMONIAN BOARD MEMBER 2.00 0.00 X 0 0 0 VANESSA VAZQUEZ BOARD MEMBER 2.00 0.00 X 0 0 0 PAUL WAXLAX BOARD MEMBER 2.00 1.00 X 0 0 0 TARA ZIMMERMAN BOARD MEMBER 2.00 0.00 X 0 0 0 KARI L ROSBECK PRESIDENT & CEO 54.00 1.00 X 194307 3598 22457 RICHARD GOLLUB CFO 47.00 3.00 X 143807 9180 24213 STEVEN L ROBERDS CHIEF SCIENTIFIC OFFICER 45.00 0.00 X 208882 0 21307 JAYE D ISHAM VP, COMMUNICATIONS STRATEGY 45.00 0.00 X 119879 0 4398 LISA M MOSS SR. DIR. DONOR RELATIONS 45.00 0.00 X 109573 0 18518 APRIL COOPER DIR. COMMUNITY PROGRAMS 45.00 0.00 X 107298 0 4722 DEAN RAGER-AGUIAR DIR. PRECLINICAL RESEARCH 45.00 0.00 X 147083 0 21593 1030829 12778 117208 7 0 1 0 PSYCHOGENICS INC 20 GRAMERCY PARK SOUTH NEW YORK NY 10003 RESEARCH LAB SERVICES 601033 VAN ANDEL RESEARCH INSTITUTE 333 BOSTWICK AVE NE GRAND RAPIDS MI 49503 LABORATORY SERVICES 144738 CAVAROCCHI RUSCIO DENNIS AND ASSOCIATES 600 MARYLAND AVE SW WASHINGTON DC 20024 GOVERNMENT RELATIONS 113547 GRAHAM-PELTON CONSULTING 39 BEECHWOOD ROAD SUMMIT NJ 07901 DEVELOPMENT STRATEGY 109122 4 28836 4208 1192939 330875 371820 2416333 91027 4345011 CONTRACT REVENUE 900099 791210 791210 CONFERENCE REVENUE 900099 1475 1475 792685 11188 11188 87545 86963 582 582 582 52486 1192939 51504 982 982 CHANGE INT-ENDOWMENT 900099 -22284 -22284 -22284 5128164 791210 0 -8057 633006 633006 52782 52782 667009 335572 232186 99251 1374953 906889 115728 352336 39528 25520 2182 11826 162832 103675 11963 47194 138470 83373 20023 35074 19257 6482 11733 1042 27285 27285 112980 112980 376676 192073 6480 178123 207621 97144 5374 105103 169610 110221 26682 32707 119343 71577 17454 30312 109888 100966 664 8258 63378 53553 9749 76 70346 47126 6541 16679 8049 4833 1184 2032 PRECLINICAL CONSORTIUM 815227 815227 NHD BIOSAMPLE REPOSIT 208188 208188 DUES AND SUBSCRIPTIONS 40684 20749 19935 MISCELLANEOUS EXPENSES 5078 2213 1226 1639 5422190 3984149 496454 941587 X 66240 33120 0 33120 809370 1097500 1227121 1238876 2033626 1578285 10243 113491 251370 392003 735604 225237 80221 510367 27556 5911809 6727351 10351316 11657873 532853 435478 54700 422226 7176 1337608 594729 2195312 X 5661364 5954477 4095223 3508084 9756587 9462561 10351316 11657873 -294026 0 X 0 1 X 1 0 X 4517826 3949953 4105376 5527543 4345011 22445709 4517826 3949953 4105376 5527543 4345011 22445709 3416325 19029384 15431 10393 13784 27444 11188 78240 44490 44490 1683 642 1243 714 4282 22572721 7774339 0.84300 0.82340 X SCHEDULE A, PART II, LINE 10, EXPLANATION OF OTHER INCOME: INCOME FROM ACTIVITIES NOT REGULARLY CARRIED ON - 2016 AMOUNT: $ 1,683. 2017 AMOUNT: $ 642. 2018 AMOUNT: $ 1,243. 2019 AMOUNT: $ 714. 2020 AMOUNT: $ 0. RESTRICTED RESTRICTED RESTRICTED RESTRICTED RESTRICTED RESTRICTED RESTRICTED RESTRICTED 895 122119 123014 5350680 5473694 423685 105921 0 0 416065 450555 445393 423685 1735698 2603547 146199 149340 165226 123014 583779 104016 112639 111348 105921 433924 650886 1797 823 2491 895 6006 5915805 54645 416431 463721 29639 5893521 5193934 78962 959121 288356 27856 5915805 5655555 82346 -439321 50000 54646 5193934 5293989 92635 739072 403000 67141 5655555 4763517 58749 525868 54145 5293989 0.85080 0.14920 0 1 1 449272 30632 418640 191620 109910 81710 94712 84695 10017 510367 INTEREST IN NET ASSETS OF AFFILIATE 5893521 OPERATING LEASE RIGHT-OF-USE ASSET 833830 6727351 DEFERRED RENT & LEASE INCENTIVE 1336433 DUE TO AFFILIATE 1175 1337608 5210068 81904 81904 5128164 0 5128164 5504094 81904 81904 5422190 0 5422190 PART V, LINE 4: THE ALLIANCE'S ENDOWMENTS CONSIST OF TWO FUNDS ESTABLISHED FOR DIFFERENT PURPOSES. THE ALLIANCE'S ENDOWMENT INCLUDE ONE TRADITIONAL DONOR-RESTRICTED ENDOWMENT FUNDS AND ONE BOARD-DESIGNATED ENDOWMENT FUND. THE BOARD-DESIGNATED ENDOWMENT FUND SOLELY CONSISTS OF THE ENDOWMENT FUND'S UNRESTRICTED NET ASSET BALANCE. 1 EUROPE (INCLUDING ICELAND & GREENLAND) 0 0 GRANTMAKING 34032 MIDDLE EAST AND NORTH AFRICA 0 0 GRANTMAKING 18750 EUROPE (INCLUDING ICELAND & GREENLAND) 0 0 PROGRAM SERVICE ACTIVITIES PRECLINICAL RESEARCH STUDIES 58944 0 0 0 0 0 0 111726 0 111726 MIDDLE EAST AND NORTH AFRICA RESEARCH ON TUBEROUS SCLEROSIS COMPLEX 18750 WIRE EUROPE (INCLUDING ICELAND & GREENLAND) - ALBANIA, ANDORRA, AUSTRIA, BELGIUM PROGRAMMATIC SUPPORT 34032 WIRE 0 2 0 0 0 0 0 0 PART I, LINE 2: GRANTEE ORGANIZATIONS ARE EXPECTED TO FILE ANNUAL PROGRESS REPORTS TO OUTLINED GRANT GOALS AND MILESTONES. THESE REPORTS ARE REVIEWED BY A COMMITTEE OF PEERS. THIS COMMITTEE MAKES DETERMINATIONS BASED ON QUALITY OF WORK TO GOALS AND IF THE GRANTEE WILL CONTINUE TO RECEIVE FUNDING. A FINAL WRITTEN AND FINANCIAL REPORT IS REQUIRED OF ALL GRANTEES. PART III ACCOUNTING METHOD: VIRTUAL WALKS 660000 626454 33546 33308 58 180 COMEDY FOR A CURE 179147 175292 3855 756 137 1979 9 406278 391193 15085 4129 8091 2739 127 1245425 1192939 52486 38193 8091 2934 2286 51504 982 1 BRIGHAM & WOMAN'S HOSPITAL 75 SAINT FRANCIS STREET BOSTON MA 02115 042312909 501(C)(3) 55436 RESEARCH GRANTS UNIVERSITY OF TEXAS SOUTHWESTERN MEDICAL CENTER 5323 HARRY HINES BLVD DALLAS TX 75287 756002868 501(C)(3) 56250 RESEARCH GRANTS YALE UNIVERSITY POBOX 1873 NEW HAVEN CT 06508 060646973 501(C)(3) 56011 RESEARCH GRANTS AMERICAN THORACIC SOCIETY 25 BROADWAY 18TH FLOOR NEW YORK NY 10004 061548706 501(C)(3) 5500 RESEARCH GRANTS CHILDREN'S HOSPITAL MEDICAL CENTER SPONSORED PRGS ACTG ML 4900 3333 BURNET AVE CINCINNATI OH 45229 310833936 501(C)(3) 50000 CLINICAL RESEARCH GRANTS NATIONAL ORGANIZATION FOR RARE DISORDERS 55 KENOSIA AVENUE DANBURY CT 06810 133223946 501(C)(3) 65000 TSC TRAVEL AND LODGING ASSISTANCE PROGRAM NORTHWESTERN UNIVERSITY 7TH FLOOR RUBLOFF BUILDING CHICAGO IL 60611 362167818 501(C)(3) 75001 RESEARCH GRANTS CHILDREN'S HOSPITAL CORPORATION 300 LONGWOOD AVENUE BOSTON MA 02115 042774441 501(C)(3) 150001 RESEARCH GRANTS BOSTON CHILDREN'S HOSPITAL 300 LONGWOOD AVENUE BOSTON MA 02115 042774441 501(C)(3) 57409 CLINICAL RESEARCH GRANT UNIVERSITY OF CALIFORNIA BERKELEY 1608 FOURTH ST STE 220 BERKELEY CA 94710 946002123 501(C)(3) 9375 RESEARCH GRANT UNIVERSITY OF ALABAMA 1720 2ND AVE SOUTH AB990 BIRMINGHAM AL 35294 636005396 501(C)(3) 15533 CLINICAL RESEARCH GRANT UNIVERSITY OF TENNESSEE HEALTH SCIENCE CTR 910 MADISON AVE STE 823 MEMPHIS TN 38103 626001636 501(C)(3) 18511 RESEARCH GRANT VANDERBILT UNIVERSITY 1161 21ST AVE SOUTH NASHVILLE TN 37232 352528741 501(C)(3) 9375 RESEARCH GRANT 13 0 PART I, LINE 2: THE TSC ALLIANCE HAS FUNDED MORE THAN $27.7 MILLION IN RESEARCH ON TSC SINCE 1984. DIRECTED BY STEVEN L. ROBERDS, PH.D, CHIEF SCIENTIFIC OFFICER, THE TSC ALLIANCE RESEARCH GRANTS PROGRAM FUNDS RESEARCH FOCUSED ON TSC WITH PRIORITIES SET BY THE RESEARCHERS TOGETHER WITH THE TSC ALLIANCE. COLLABORATIONS BETWEEN BASIC AND CLINICAL RESEARCHERS ARE ENCOURAGED AND FOSTERED, AND THE TSC ALLIANCE IS WORKING TO INCREASE FUNDING FOR RESEARCH ON TSC. THROUGH THE TSC ALLIANCE RESEARCH GRANTS PROGRAM, APPLICATIONS CAN BE SUBMITTED FOR: POSTDOCTORAL FELLOWSHIPS AND TSC RESEARCH GRANTS. GRANTS ARE REVIEWED IN A THREE-STEP PROCESS: 1. A GRANT REVIEW COMMITTEE COMPOSED OF INDIVIDUALS KNOWLEDGEABLE ABOUT THE CLINICAL AND BASIC COMPONENTS OF TSC AS WELL AS CONSUMERS REVIEW ALL GRANT APPLICATIONS FOR SCIENTIFIC MERIT, RELEVANCY TO THE FUNDING PRIORITIES OF THE ORGANIZATION AND WITH A FOCUS ON UNDERSTANDING THE MECHANISMS OF TSC AND/OR THE DEVELOPMENT OF TREATMENTS AND THERAPIES FOR THE MANIFESTATIONS OF THE DISEASE. 2. THE SCIENCE AND MEDICAL COMMITTEE OF THE BOARD OF DIRECTORS THEN REVIEWS THE GRANT REVIEW COMMITTEE'S CONCLUSIONS AND MAKES FUNDING RECOMMENDATIONS TO THE BOARD OF DIRECTORS. 3. THE BOARD OF DIRECTORS THEN REVIEWS THE RECOMMENDATIONS OF THE SCIENCE AND MEDICAL COMMITTEE AND MAKES FINAL APPROVAL FOR THE FUNDING OF GRANTS. X X X X 0 0 0 1 0 1 0 0 0 KARI L ROSBECK PRESIDENT & CEO 176975 3277 17191 318 141 3 6045 112 17047 316 217399 4026 0 0 RICHARD GOLLUB CFO 131082 8367 12338 788 387 25 4504 288 19255 1229 167566 10697 0 0 STEVEN L ROBERDS CHIEF SCIENTIFIC OFFICER 190616 0 17998 0 268 0 6407 0 15963 0 231252 0 0 0 DEAN RAGER-AGUIAR DIR. PRECLINICAL RESEARCH 140752 0 6188 0 143 0 4674 0 17982 0 169739 0 0 0 PART I, LINE 5 KARI LUTHER ROSBECK, RICHARD GOLLUB, STEVE ROBERDS, DEAN AGUIAR, JAYE ISHAM, LISA MOSS AND APRIL COOPER ALL HAVE INCENTIVE COMPENSATION EQUAL TO A PERCENTAGE OF THEIR SALARIES BASED ON KEY PERFORMANCE OBJECTIVES AS ESTABLISHED BY THE COMPENSATION COMMITTEE. PART I, LINE 6 KARI LUTHER ROSBECK, RICHARD GOLLUB, STEVE ROBERDS, DEAN AGUIAR, JAYE ISHAM, LISA MOSS AND APRIL COOPER ALL HAVE INCENTIVE COMPENSATION EQUAL TO A PERCENTAGE OF THEIR SALARIES BASED ON KEY PERFORMANCE OBJECTIVES AS ESTABLISHED BY THE COMPENSATION COMMITTEE. X 422 FMV X 6 87545 FMV X 2 3060 FMV 0 0 1 0 FORM 990, PART III, LINE 4A (CONT'D): IN 2012, THE TSC ALLIANCE HELPED CREATE THE TSC CLINICAL RESEARCH CONSORTIUM IN PARTNERSHIP WITH INVESTIGATORS RUNNING CLINICAL STUDIES TO ENSURE CLINICAL RESEARCH IN TSC IS AS EFFICIENT AND EFFECTIVE AS POSSIBLE. SINCE THEN, TSC CLINICAL RESEARCH CONSORTIUM INVESTIGATORS HAVE BEEN AWARDED MORE THAN $35 MILLION BY THE NATIONAL INSTITUTES OF HEALTH (NIH) THROUGH COMPETITIVE GRANT PROCESSES. TSC ALLIANCE PERSONNEL SERVE ON THE LEADERSHIP TEAM FOR THE CONSORTIUM, ACTIVELY TRACK ENROLLMENT, AND RAISE COMMUNITY AWARENESS TO HELP IDENTIFY POTENTIAL PARTICIPANTS FOR CLINICAL STUDIES. TSC ALLIANCE ALSO PROVIDES SUPPLEMENTAL FINANCIAL SUPPORT TO ACCELERATE OR EXPAND NIH-FUNDED STUDIES. THE PREVENT TRIAL - PREVENTING EPILEPSY USING VIGABATRIN IN INFANTS WITH TSC - IS THE FIRST PREVENTATIVE TRIAL IN THE UNITED STATES FOR ANY FORM OF EPILEPSY AND HAS COMPLETED ENROLLMENT OF 86 PARTICIPANTS. WE EXPECT THE FIRST RESULTS IN DECEMBER 2021 AND FINAL RESULTS IN LATE 2022 OR EARLY 2023. THIS TRIAL BUILDS DIRECTLY UPON THE CLINICAL RESEARCH CONSORTIUM'S FIRST CLINICAL STUDY, WHICH IDENTIFIED SPECIFIC EEG CHANGES IN INFANTS WITH TSC PRIOR TO THE ONSET OF SEIZURES. IN THE PREVENT TRIAL, INFANTS WITH TSC RECEIVE REGULAR EEG MONITORING, AND THOSE WHO DEVELOP EEG ABNORMALITIES ARE PLACED ON EITHER VIGABATRIN OR PLACEBO. ANY INFANT WHO SUBSEQUENTLY DEVELOPS CLINICAL SEIZURES IS IMMEDIATELY PLACED ON STANDARD TREATMENT. THIS TRIAL WILL DETERMINE WHETHER TREATMENT WITH VIGABATRIN PRIOR TO THE ONSET OF CLINICAL SEIZURES IN TSC IS BENEFICIAL TO CHILDREN'S DEVELOPMENTAL AND NEUROLOGIC OUTCOMES. MORE DETAILS ABOUT THIS STUDY ARE ONLINE AT WWW.CLINICALTRIALS.GOV/CT2/SHOW/NCT02849457. IMMEDIATELY AFTER THE PREVENT TRIAL ENROLLMENT COMPLETED, A SIMILAR CLINICAL TRIAL BEGAN ENROLLING NEWBORNS WITH TSC UTILIZING A SIMILAR PROTOCOL BUT TESTING THE EFFECTS OF SIROLIMUS INSTEAD OF VIGABATRIN. THE STOPPING TSC ONSET AND PROGRESSION 2 (STOP-2) TRIAL OPENED AT CINCINNATI CHILDREN'S HOSPITAL MEDICAL CENTER, AND THE TSC ALLIANCE IS CONTRIBUTING $200,000 TO ENABLE THE TRIAL TO BE EXPANDED TO ADDITIONAL SITES ACROSS THE COUNTRY IN 2021. TSC ALLIANCE IS ALSO PROVIDING SUPPLEMENTAL FUNDING AND COLLECTING BLOOD SAMPLES FOR THE DEVELOPMENTAL SYNAPTOPATHIES CONSORTIUM (DSC), AN NIH-FUNDED PROJECT THAT INCLUDES STUDIES OF TSC AND THE RELATED RARE DISORDERS PHELAN-MCDERMID SYNDROME AND PTEN HAMARTOMA SYNDROME. THESE THREE RARE DISEASES SEEM TO AFFECT CERTAIN SHARED PATHWAYS INFLUENCING THE DEVELOPMENT OF BRAIN CONNECTIONS, OR SYNAPSES. RESEARCHERS IN THIS STUDY ARE TRYING TO FIND EARLIER SIGNS OF AUTISM SPECTRUM DISORDER (ASD) AND INTELLECTUAL DISABILITY (ID) TO GAIN A BETTER UNDERSTANDING OF ASD/ID IN INDIVIDUALS WITH TSC AND ENABLE EFFECTIVE TREATMENTS AND INTERVENTIONS FOR ASD/ID TO BE FOUND. THE DSC WAS RENEWED LAST YEAR FOR A SECOND 5-YEAR FUNDING PERIOD. TSC ALLIANCE FUNDS A PORTION OF CLINICAL RESEARCH COORDINATORS' SALARIES AT 5 SITES AND WILL BEGIN FUNDING A 2-YEAR CLINICAL FELLOWSHIP IN TSC BEGINNING 2021. IN ADDITION TO SPECIFIC COLLABORATIVE PROJECTS DETAILED ABOVE, THE TSC ALLIANCE STIMULATES COLLABORATION AND INNOVATION THROUGH CONVENING EXPERTS AND STAKEHOLDERS WITHIN AND OUTSIDE THE TSC FIELD. THE FIRST INNOVATION WORKSHOP WAS HELD IN 2020 AND FOCUSED ON NEWBORN SCREENING. THIS WORKSHOP'S GOALS INCLUDED: DEFINE THE CRITICAL PATH - OR RATE-LIMITING STEPS - OF DEVELOPING A SCREENING ASSAY TO ENABLE NOMINATION OF TSC TO THE RECOMMENDED UNIFORM SCREENING PANEL; SPECIFY THE IMMEDIATE NEXT STEPS NEEDED ALONG THE CRITICAL PATH; RECOMMEND COMPONENTS OF A FUNDING OPPORTUNITY ANNOUNCEMENT TO ELICIT THE BEST IDEAS AND APPLICANTS TO IMPLEMENT THOSE NEXT STEPS; AND ENABLE TSC ALLIANCE TO FUND THE MOST MERITORIOUS APPLICATIONS WITHIN 100 DAYS AFTER THE WORKSHOP. ORIGINALLY, THE TSC ALLIANCE HAD PLANNED TO HOST AN IN-PERSON WORKSHOP TO BE HELD IN EARLY JUNE. HOWEVER, DUE TO THE NOVEL CORONAVIRUS PANDEMIC, THE INNOVATION WORKSHOP WAS RE-IMAGINED AS A SERIES OF VIRTUAL MEETINGS PUNCTUATED BY ONLINE DISCUSSION USING THE PLATFORM POWERNOODLE. THE FINAL CUMULATIVE DISCUSSION WILL TAKE PLACE IN EARLY JANUARY 2021 AND WILL BE FOLLOWED BY AN FOA IN THE FIRST QUARTER OF 2021. FORM 990, PART VI, SECTION A, LINE 6 MEMBERSHIP IS AVAILABLE TO ANY PERSON WHO SUBSCRIBES TO THE PURPOSES AND OBJECTIVES OF THE CORPORATION, WITHOUT REGARD TO RACE, RELIGION, GENDER, SEXUAL ORIENTATION, AGE, COLOR, NATIONAL ORIGIN OR MENTAL OR PHYSICAL HANDICAP OR DISABILITY. THERE SHALL BE NO LIMIT TO THE NUMBER OF MEMBERS IN THE CORPORATION. 1) THERE MAY BE ONE OR MORE CLASSES OF MEMBERSHIP AS DETERMINED BY THE BOARD. 2) MEMBERSHIP IS NOT TRANSFERABLE OR ASSIGNABLE. FORM 990, PART VI, SECTION A, LINE 7A THE TSC ALLIANCE IS A MEMBERSHIP-BASED ORGANIZATION, WHICH MEANS MEMBERS HELP ELECT THE BOARD OF DIRECTORS. THE TSC ALLIANCE MEMBERSHIP PROGRAM ALLOWED INDIVIDUALS TO STATE THEIR INTENT TO BE A MEMBER FOR THE PURPOSE OF GOVERNANCE. THERE WERE NO LEVELS TO MEMBERSHIP IN 2020. ANYONE CAN BE A MEMBER AT NO COST. FORM 990, PART VI, SECTION B, LINE 11B THE FORM 990 IS REVIEWED, IN DETAIL, BY THE BOARD OF DIRECTORS' AUDIT COMMITTEE. RECOMMENDATIONS ARE MADE BY THE COMMITTEE MEMBERS FOR ANY CHANGES/EDITS/ADDITIONS. AFTER THOSE HAVE BEEN INCORPORATED, THE AUDIT COMMITTEE VOTES WHETHER TO APPROVE AND THEN FORWARD THE 990 TO THE FINANCE AND EXECUTIVE COMMITTEES. THE FINANCE AND EXECUTIVE COMMITTEES PERFORM THE FINAL REVIEW AND THEN VOTE WHETHER TO APPROVE ON BEHALF OF THE BOARD OF DIRECTORS. A COPY OF THE APPROVED 990 IS SHARED WITH THE ENTIRE BOARD PRIOR TO ITS FILING WITH THE IRS. FORM 990, PART VI, SECTION B, LINE 12C ANNUALLY EACH MEMBER OF THE BOARD OF DIRECTORS WILL RECEIVE NOTICE OF THE ORGANIZATION'S CONFLICT OF INTEREST STATEMENT. EACH MEMBER WILL BE PROVIDED WITH A STATEMENT TO MAKE DISCLOSURE OF ANY POTENTIAL CONFLICT OF INTEREST. IF DURING THE COURSE OF THE YEAR A POTENTIAL CONFLICT OF INTEREST ARISES THAT HAS NOT PREVIOUSLY BEEN DISCLOSED, THE BOARD MEMBER WILL MAKE WRITTEN NOTICE OF A POTENTIAL CONFLICT OF INTEREST AND RECUSE HIMSELF OR HERSELF FROM ANY DISCUSSIONS AND VOTES IN CONNECTION WITH THE ISSUE IDENTIFIED. ANY TIME A MEMBER IS RECUSED FROM DISCUSSION ON AN ISSUE, THE MINUTES OF COMMITTEE MEETING AND BOARD MEETING WILL DULY RECORD SUCH ACTIONS. THE FOLLOWING POTENTIAL CONFLICTS OF INTEREST WERE DISCLOSED FOR 2020: BOARD MEMBER MUSTAFA SAHIN, PH.D., M.D., IS EMPLOYED AT BOSTON CHILDREN'S HOSPITAL, WHICH RECEIVED $132,409 IN GRANTS AND $13,935 IN FEES FOR SERVCIES FOR PARTICIPATION IN THE TSC NATURAL HISTORY DATABASE. BOARD MEMBER DARCY KRUEGER, MD, MPA, IS EMPLOYED AT THE CINCINNATI CHILDREN'S HOSPITAL MEDICAL CENTER, WHICH RECEIVED A $50,000 GRANT PAYMENT FOR HIS WORK ON STOP-2 AND $26,335 IN FEES FOR SERVICES FOR PARTICIPATION IN THE TSC NATURAL HISTORY DATABASE. FORM 990, PART VI, SECTION B, LINE 15 THE COMPENSATION COMMITTEE REVIEWS AND APPROVES THE SALARIES OF THE PRESIDENT/CEO, CHIEF SCIENTIFIC OFFICER, CONTROLLER & CFO, AND ANY EMPLOYEE APPEARING ON THE FORM 990, IN ACCORDANCE WITH THE TSC ALLIANCE BYLAWS. SUCH REVIEW AND APPROVAL OCCURS INITIALLY UPON HIRING, UPON ANNUAL REVIEWS AND WHENEVER MODIFIED. THE ORGANIZATION'S EXECUTIVE REMUNERATION HAS BEEN STRUCTURED TO ENSURE THAT IT: IS REASONABLE; PROVIDES A COMPETITIVE COMPENSATION PROGRAM TO RETAIN, ATTRACT AND REWARD KEY EMPLOYEES AND ACHIEVES CLEAR ALIGNMENT BETWEEN TOTAL REMUNERATION AND DELIVERED BUSINESS AND PERSONAL PERFORMANCE OVER THE SHORT AND LONG-TERMS. THE COMPENSATION IS REVIEWED BY THE COMPENSATION COMMITTEE TO ENSURE: - COMPARABILITY, - PROPER REVIEW, AND - SUBSTANTIATION IN SETTING THE COMPENSATION. FORM 990, PART VI, SECTION C, LINE 19 ALL GOVERNING DOCUMENTS, CONFLICT OF INTEREST POLICY AND FINANCIAL STATEMENTS ARE AVAILABLE UPON WRITTEN REQUEST. TSC ALLIANCE ENDOWMENT FUND INC 8737 COLESVILLE ROAD SUITE 400 SILVER SPRING MD 20910 521926919 SUPPORT ORG MD 501(C)(3) LINE 12B, II N/A 0 0 0 1 0 0 0 0 0 0 0 0 0 0 1 1 0 0 0 0 TSC ALLIANCE ENDOWMENT FUND INC C 330875 CASH