2024-09-24T12:54:15-05:00 2023-12-31 470900880 CALIBRE CPA GROUP PLLC 7501 WISCONSIN AVENUE SUITE 1200 WEST BETHESDA MD 20814 990 2023-01-01 953018799 NATIONAL TUBEROUS SCLEROSIS ASSOCIATION NATI 3015629890 8737 COLLESVILLE ROAD 400 SILVER SPRING MD 20910 KARI L ROSBECK PRESIDENT & CEO 3015629890 2024-09-17 1 1 KRISTIN A JACQUELIN CPA P01325865 2023319880 2024-09-24 2023 2024-08-08 14:25:15Z X TSC ALLIANCE KARI L ROSBECK 8737 COLLESVILLE ROAD 400 SILVER SPRING MD 20910 10180515 0 X WWW.TSCALLIANCE.ORG X 1975 CA TO IMPROVE QUALITY OF LIFE FOR EVERYONE AFFECTED BY TUBEROUS SCLEROSIS COMPLEX BY CATALYZING NEW TREATMENTS, DRIVING RESEARCH TOWARD A CURE AND EXPANDING ACCESS TO LIFELONG SUPPORT. 30 30 26 2234 0 0 7016851 6747948 2720317 2815353 2636 36732 -830262 416350 8909542 10016383 1032155 845292 0 0 2896346 3024376 0 0 1150291 4135183 4597980 8063684 8467648 845858 1548735 14827937 16085768 2373091 2088420 12454846 13997348 X TO IMPROVE QUALITY OF LIFE FOR EVERYONE AFFECTED BY TUBEROUS SCLEROSIS COMPLEX BY CATALYZING NEW TREATMENTS, DRIVING RESEARCH TOWARD A CURE AND EXPANDING ACCESS TO LIFELONG SUPPORT. 0 0 5022281 845292 2621353 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 MORE THAN $37 MILLION INTO TSC RESEARCH PROJECTS THROUGH GRANTS AND CONTRACTS: $20.6 MILLION IN RESEARCH GRANTS AND POSTDOCTORAL FELLOWSHIPS, $5.3 MILLION INTO THE NATURAL HISTORY DATABASE AND BIOSAMPLE REPOSITORY, $9.6 MILLION INTO THE PRECLINICAL CONSORTIUM, AND $1.6 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 TSCALLIANCE.ORG/GRANTS.IN 2023, THE TSC ALLIANCE HOSTED OUR BIENNIAL INTERNATIONAL TSC RESEARCH CONFERENCE: FUELING THE FUTURE. FIFTEEN PERCENT OF ATTENDEES SELF-IDENTIFIED AS MEMBERS OF GROUPS HISTORICALLY UNDERREPRESENTED IN BIOMEDICAL RESEARCH. THE CONFERENCE FEATURED AN EARLY CAREER RESEARCH SYMPOSIUM, A KEYNOTE SPEECH FROM DR. MARTINA BEBIN, 30 ORAL PRESENTATIONS, 59 POSTERS, A TSC INTERNATIONAL WORKSHOP, AND A COMBINED CLOSING SESSION WITH TSC & LAM REGIONAL CONFERENCE PARTICIPANTS. THE CONFERENCE ALSO FEATURED FOUR BREAKOUT WORKING GROUP SESSIONS (TRANSITION FROM PEDIATRIC TO ADULT, NEURODEVELOPMENT AND EARLY INTERVENTION, CELLULAR ENERGETICS AND METABOLISM, AND BIG DATA AND SINGLE CELL APPROACHES/ANALYSIS) TO ALLOW TIME FOR ATTENDEES TO MEET WITH OTHERS IN THEIR FIELD AND WORK TOWARD SOLUTIONS FOR CURRENT ISSUES IN THEIR AREA.IMPLEMENTED IN 2006, THE TSC NATURAL HISTORY DATABASE (NHD) CAPTURES CLINICAL DATA TO DOCUMENT THE IMPACT OF THE DISEASE ON A PERSON'S HEALTH OVER HIS/HER 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 2023, 2,678 PEOPLE WITH TSC WERE ENROLLED IN THE PROJECT FROM AMONG 22 TSC CLINIC 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 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 10 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 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. AS OF DECEMBER 31, 2023, THE TSC BIOSAMPLE REPOSITORY HAS ACQUIRED 2,654 BIOSAMPLES: 1012 BLOOD SAMPLES FROM INDIVIDUALS WITH TSC ENROLLED IN THE NHD, 452 BUCCAL SAMPLES FROM INDIVIDUALS WITH TSC ENROLLED IN THE NHD, 197 TISSUE SAMPLES FROM INDIVIDUALS WITH TSC ENROLLED IN THE NHD, 591 BLOOD SAMPLES FROM THE TSC CLINICAL RESEARCH CONSORTIUM COLLABORATIVE PROJECTS, 389 DNA SAMPLES FROM THE TSC AUTISM CENTER OF EXCELLENCE NETWORK AUTISM BIOMARKER STUDY, AND 10 CONTROL SAMPLES. THE ORGANIZATION CONTINUES 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. SINCE THE INCEPTION OF OUR MOBILE PHLEBOTOMY INITIATIVE, WE HAVE ACQUIRED 473 BLOOD SAMPLES VIA MOBILE PHLEBOTOMY. TO ENCOURAGE AND ENABLE MORE RESEARCHERS TO UTILIZE BIOSAMPLES, THE TSC ALLIANCE AWARDED THREE SEED GRANTS IN 2023, TOTALING $60,000, TO LABS THAT SUBMITTED MERITORIOUS AND INNOVATIVE IDEAS FOR RESEARCH ON TSC BIOSAMPLES.THE TSC ALLIANCE'S WHOLE-GENOME SEQUENCING (WGS) INITIATIVE USING DNA FROM BLOOD SAMPLES IN OUR BIOSAMPLE REPOSITORY SEQUENCED AN ADDITIONAL 72 SAMPLES IN 2023, TOTALING 115 SAMPLES SINCE INCEPTION. THIS INITIATIVE SUPPORTS CLINICAL VALIDATION OF VARIANTS FOUND VIA WGS IN EITHER THE TSC1 OR TSC2 GENE, AND GENETIC RESULTS ARE OFFERED BACK TO PARTICIPANTS ALONG WITH A GENETIC COUNSELING SESSION FREE OF CHARGE TO THE FAMILY TO HELP THEM BETTER UNDERSTAND THEIR UNIQUE TSC DIAGNOSIS AND PROVIDE VALUABLE INFORMATION FOR FUTURE DECISION MAKING SUCH AS FAMILY PLANNING.IN DECEMBER 2021, THE TSC ALLIANCE ADDED THE TSC SELF-REPORT PORTAL TO THE TSC NATURAL HISTORY DATABASE. THIS PORTAL PERMITS THE COLLECTION OF PATIENT-REPORTED OUTCOMES ON HOW TSC AFFECTS INDIVIDUALS AND FAMILIES, WHICH WILL COMPLEMENT MEDICAL DATA IN THE NATURAL HISTORY DATABASE. THE PURPOSE OF THIS INITIATIVE IS TO HELP THE TSC ALLIANCE AND TSC RESEARCHERS BETTER UNDERSTAND THE PERSPECTIVE OF THOSE AFFECTED BY TSC TO DEVELOP TOOLS TO MEASURE IMPROVEMENT IN AREAS MOST IMPORTANT TO THE TSC COMMUNITY. EVENTUALLY, THESE MEASUREMENTS CAN BE USED TO IDENTIFY ENDPOINTS FOR CLINICAL TRIALS AND EVIDENCE-BASED GUIDELINES FOR TREATMENT.ONE OF THE MOST IMPACTFUL ASPECTS OF TSC ON THE QUALITY OF LIFE FOR PEOPLE LIVING WITH TSC IS TSC-ASSOCIATED NEUROPSYCHIATRIC DISORDERS (TAND). THEREFORE, THE TSC ALLIANCE'S FIRST USE OF THE PORTAL IS THROUGH COLLABORATION WITH THE TANDEM PROJECT (EMPOWERING FAMILIES THROUGH TECHNOLOGY: A MOBILE-HEALTH PROJECT TO REDUCE THE TAND IDENTIFICATION AND TREATMENT GAP) BY ALLOWING THE COMMUNITY TO COMPLETE THE SELF-QUANTIFIED TAND CHECKLIST (TAND-SQ). AT THE END OF 2023, 105 INDIVIDUALS WERE ENROLLED THROUGH THE SELF-REPORT PORTAL.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 TAND. IN 2022, THE TSC ALLIANCE ENTERED INTO A PARTNERSHIP WITH THE LAM FOUNDATION TO ADD LAM MOUSE MODELS TO THE PRECLINICAL CONSORTIUM. 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 F L7-CRE-TSC2 MICE ON BEHALF OF THE PRECLINICAL CONSORTIUM. 895227 SUPPORT SERVICES. SEE SCHEDULE O FOR FULL PROGRAM DESCRIPTION. 554030 PUBLIC HEALTH. SEE SCHEDULE O FOR FULL PROGRAM DESCRIPTION. 287855 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 ALABAMA, MISSOURI, AND MARYLAND, RESULTING IN AT LEAST $1.25 MILLION 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 2023. ADVOCATES MET WITH MORE THAN 322 CONGRESSIONAL OFFICES VIRTUALLY AND IN PERSON TO ASK FOR $10 MILLION IN FUNDING FOR THE TSCRP. THE HOUSE TSCRP DEAR COLLEAGUE LETTER SPONSORED BY REPRESENTATIVES FITZPATRICK (R-PA) AND RASKIN (D-MD) CLOSED WITH 191SIGNERS. THE SENATE LETTER SPONSORED BY SENATORS CRAMER (R-ND) AND HEINRICH (D-NM) CLOSED WITH 41 SIGNERS. TOTAL FUNDING FOR THE TSCRP HAS BEEN $113 MILLION SINCE 2002, INCLUDING A $10 MILLION APPROPRIATION FOR FY234. 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; TWO TSCRP AWARDS IN FY2012 AND FY2015 THAT ENABLED GENERATION OF A POTENTIAL APPROACH FOR GENE THERAPY OF TSC, WHICH HAS SHOWN PROMISING RESULTS IN A MOUSE MODEL OF TSC TUMORS IN THE BRAIN; 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 THAT LED THE THIRD FDA-APPROVED DRUG TO TREAT TSC IN APRIL 2022; AND A CLINICAL RESEARCH NETWORK THAT 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.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 TSC ALLIANCE OF ISRAEL, TS CANADA ST, TSC ALLIANCE OF MEXICO, TSC ALLIANCE FOUNDATION (IN THAILAND), HUNGARIAN FOUNDATION FOR TUBEROUS SCLEROSIS, AND TSC ALLIANCE OF INDIA. THE TSC ALLIANCE RECOGNIZES 13 TSC CLINICS IN GLOBAL ALLIANCE COUNTRIES. 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 EXHIBITED AT THE AMERICAN EPILEPSY SOCIETY ANNUAL MEETING. STAFF MEMBERS AND A VOLUNTEER SHARED EDUCATIONAL MATERIALS WITH ATTENDEES WHO VISITED THE BOOTH. STAFF ALSO PARTICIPATED IN THE TSC SPECIAL INTEREST GROUP SESSION, AND THE TSC ALLIANCE HOSTED MORE THAN 100 PEOPLE AT A RECEPTION WITH PRESENTATIONS BY PARENTS OF A CHILD WITH TSC AND BY DR. ANGELIQUE BORDEY, WHO DESCRIBED HOW TSC ALLIANCE HAS IMPACTED HER RESEARCH. THE TSC ALLIANCE PARTICIPATED IN OR PRESENTED AT 26 PROFESSIONAL MEETINGS IN 2023 INCLUDING PRETOLA COURSE, RESEARCH AMERICA ANNUAL ADVOCACY AWARDS, WOMEN AT BRIDGE LEADERSHIP SPEAKING SERIES, INSITRO TOWN HALL, RARE DISEASE INNOVATION AND PARTNERING SUMMIT, ANYA'S ACCELERATOR WORKSHOP, NORD, TSC ALLIANCE MEXICO BRIEFING, EAN CONGRESSIONAL BRIEFING, ATS ANNUAL MEETING, INTERAGENCY COLLABORATIVE TO ADVANCE RESEARCH IN EPILEPSY (ICARE), RARE DISEASES INTERNATIONAL WORLD HEALTH ASSEMBLY, UCP ANNUAL CONFERENCE, MARINUS TOWN HALL, TANDEM MINI SYMPOSIUM, ETSC WORKSHOP, NINDS NONPROFIT FORUM, CRISIS INTERVENTION TRAINING INTERNATIONAL, TSCI WORKSHOP, TSC REPRODUCTIVE & PERINATAL TASK FORCE MEETING, LGS MEETING, GLOBAL GENES RARE SUMMIT, PCORI ANNUAL MEETING, APERTURA TOWN HALL, RDCRN FALL MEETING, AES, AND EAN BRIEFING. 287855 6759393 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 0 0 0 0 0 1 0 0 0 1 33 0 1 26 1 0 0 0 0 0 1 1 0 0 0 0 0 0 X 30 30 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 TSC ALLIANCE 3015629890 8737 COLLESVILLE ROAD 400 SILVER SPRING MD 20910 WILLIAM JOSEPH CHAIR 5.00 X X 0 0 0 PAUL WAXLAX VICE-CHAIR 5.00 1.00 X X 0 0 0 BRITTANY SMITH TREASURER 5.00 1.00 X X 0 0 0 LESLEY HOLMES SECRETARY 2.00 X X 0 0 0 PETER CRINO IMMEDIATE PAST CHAIR 2.00 X 0 0 0 DEREK BAUER BOARD MEMBER 2.00 X 0 0 0 JULIE BLUM BOARD MEMBER 2.00 X 0 0 0 MATT BOLGER BOARD MEMBER 2.00 X 0 0 0 CHIP BURKHALTER BOARD MEMBER 2.00 X 0 0 0 LISA CARLTON BOARD MEMBER 2.00 X 0 0 0 DAVID COIT BOARD MEMBER 2.00 X 0 0 0 MARK CARROLL BOARD MEMBER 2.00 X 0 0 0 JULIAN GANGOLLI BOARD MEMBER 2.00 X 0 0 0 TANJALA GIPSON BOARD MEMBER 2.00 X 0 0 0 JONATHAN GOLDSTEIN BOARD MEMBER 2.00 X 0 0 0 STEVEN GOLDSTEIN BOARD MEMBER 2.00 X 0 0 0 ROB GRANDIA BOARD MEMBER 2.00 X 0 0 0 DANA HOLINKA BOARD MEMBER 2.00 X 0 0 0 SHAFALI JESTE BOARD MEMBER 2.00 X 0 0 0 JESSICA KREFTING BOARD MEMBER 2.00 X 0 0 0 DARCY KRUEGER BOARD MEMBER 2.00 X 0 0 0 PATROSKI LAWSON BOARD MEMBER 2.00 X 0 0 0 KRISTI LENGYEL BOARD MEMBER 2.00 X 0 0 0 HEATHER LENS BOARD MEMBER 2.00 X 0 0 0 LAURA MARKS BOARD MEMBER 2.00 X 0 0 0 DIANE MCSWAIN BOARD MEMBER 2.00 X 0 0 0 MUSTAFA SAHIN BOARD MEMBER 2.00 X 0 0 0 SEAN SHILLINGER BOARD MEMBER 2.00 X 0 0 0 VANESSA VAZQUEZ BOARD MEMBER 2.00 X 0 0 0 TARA ZIMMERMAN BOARD MEMBER 2.00 X 0 0 0 KARI LUTHER ROSBECK PRESIDENT & CEO 54.00 1.00 X 223746 2260 29417 CYNTHIA ARCURI CHIEF FINANCIAL OFFICER 42.00 1.00 X 176989 9315 9482 STEVEN ROBERDS CHIEF SCIENTIFIC OFFICER 45.00 X 237208 0 26563 DEAN RAGER-AGUIAR VICE PRESIDENT, TRANSLATIONAL RESEARCH 45.00 X 177725 0 28091 LISA MOSS VICE PRESIDENT, DONOR RELATIONS 45.00 X 124914 0 24144 JAYE ISHAM CHIEF OUTREACH OFFICER 45.00 X 137218 0 5426 ASHLEY POUNDERS DIRECTOR OF MEDICAL AFFAIRS 45.00 X 115372 0 13076 1193172 11575 136199 7 0 1 0 PSYCHOGENICS INC 20 GRAMERCY PARK S NEW YORK NY 10003 RESEARCH LAB SERVICES 1565754 VAN ANDEL RESEARCH INSTITUTE 333 BOSTWICK AVE NE GRAND RAPIDS MI 49503 RESEARCH LAB SERVICES 493453 CAVAROCCHI RUSCIO DENNIS & ASSOCIATES 600 MARYLAND AVE SW SUITE 220 EAST WASHINGTON DC 20024 GOVERNMENT RELATIONS 117714 3 40093 1100 1179453 572625 4954677 6747948 CONTRACT REVENUE 900099 2548976 2548976 CONFERENCE REVENUE 900099 266377 72377 194000 2815353 36732 36732 258687 1179453 164132 94555 94555 CHANGE IN INTEREST OF AFFILIATE 900099 320800 320800 995 995 321795 10016383 2621353 0 647082 696542 696542 148750 148750 703405 439278 102064 162063 1867712 1166420 271060 430232 52530 33680 5461 13389 222386 142119 24273 55994 178343 113532 20556 44255 17690 11342 2750 3598 23250 14907 3614 4729 116364 74606 18090 23668 219360 140642 34102 44616 8473 5492 9 2972 328107 172198 9714 146195 161819 80801 15642 65376 144753 91950 16592 36211 352743 294269 5315 53159 447239 404633 7811 34795 69312 52790 5255 11267 13070 8319 1511 3240 PRECLINICAL CONSORTIUM 2321442 2321442 NHD BIOSAMPLE REPOSIT 328386 328386 DUES AND SUBSCRIPTIONS 39981 13730 12520 13731 5991 3565 1625 801 8467648 6759393 557964 1150291 X 43084 21542 21542 0 1711209 1487710 694859 700650 5280316 4306013 181490 350534 277696 283821 811270 417309 463272 393961 1752986 6219095 6810093 14827937 16085768 891501 813246 285264 189493 1196326 1085681 2373091 2088420 X 5151339 6372958 7303507 7624390 12454846 13997348 14827937 16085768 1548735 -6233 0 X X 0 1 X 1 0 X 5527543 4345011 5769053 7016851 6496698 29155156 5527543 4345011 5769053 7016851 6496698 29155156 4732702 24422454 27444 11188 3809 2382 36732 81555 714 15492 995 17201 29253912 10557404 0.83480 0.82010 X RESTRICTED RESTRICTED RESTRICTED RESTRICTED RESTRICTED RESTRICTED RESTRICTED RESTRICTED 1345 187396 188741 7128616 7317357 515868 128967 0 0 423685 497465 550968 515868 1987986 2981979 123014 154139 162771 188741 628665 105921 124366 137742 128967 496996 745494 895 1127 1395 1345 4762 5500387 141816 812509 572625 60900 5821187 6711748 68423 -860036 238000 181748 5500387 5893521 132476 1053588 333813 34024 6711748 5915805 54645 416431 463721 29639 5893521 5193934 78962 959121 288356 27856 5915805 0.83606 0.15789 0.00605 0 1 1 449272 153160 296112 281726 183877 97849 80272 80272 0 393961 NET ASSETS OF AFFILIATE 5821187 OPERATING LEASE RIGHT-OF-USE ASSET 654282 DUE FROM AFFILIATE 334624 6810093 OPERATING LEASE LIABILITY 1084988 DUE FROM RELATED PARTY 693 1085681 10024868 -6233 14718 8485 10016383 0 10016383 8482366 14718 14718 8467648 0 8467648 PART V, LINE 4: THE ALLIANCE'S ENDOWMENTS CONSIST OF TWO FUNDS ESTABLISHED FOR DIFFERENT PURPOSES. THE ALLIANCE'S ENDOWMENTS INCLUDE ONE TRADITIONAL DONOR-RESTRICTED ENOWMENT FUND AND ONE BOARD-DESIGNATED ENDOWMENT FUND. THE BOARD-DESIGNATED ENDOWMENT FUND SOLELY CONSISTS OF THE ENDOWMENT FUND'S UNRESTRICTED NET ASSET BALANCE. PART XII, LINE 2D - OTHER ADJUSTMENTS: ROUNDING PART XII, LINE 4B - OTHER ADJUSTMENTS: FUNDRAISING EXPENSES INCLUDED ON PART VIII 1 EUROPE (INCLUDING ICELAND & GREENLAND) - ALBANIA, ANDORRA, AUSTRIA, BELGIUM 3 0 GRANTS TO RECIPIENTS LOCATED IN REGION RESEARCH GRANTS 148750 3 0 0 0 3 0 148750 0 148750 EUROPE (INCLUDING ICELAND & GREENLAND) - ALBANIA, ANDORRA, AUSTRIA, BELGIUM RESEARCH ON TUBEROUS SCLEROSIS COMPLEX 18750 WIRE TRANSFER 0 EUROPE (INCLUDING ICELAND & GREENLAND) - ALBANIA, ANDORRA, AUSTRIA, BELGIUM RESEARCH ON TUBEROUS SCLEROSIS COMPLEX 75000 WIRE TRANSFER 0 MIDDLE EAST AND NORTH AFRICA - ALGERIA, BAHRAIN, DJIBOUTI, EGYPT, RESEARCH 8500 WIRE TRANSFER 0 EUROPE (INCLUDING ICELAND & GREENLAND) - ALBANIA, ANDORRA, AUSTRIA, BELGIUM YOUNG INVESTIGATOR FELLOWSHIP AWARD 27500 WIRE TRANSFER 0 0 4 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. WALK-RUN-RIDE EVENTS 789897 789897 44986 6147 8314 12797 COMEDY FOR A CURE 464844 389556 75288 1128 28633 44988 2969 8 183399 183399 2478 8699 2993 1438140 1179453 258687 48592 43479 53302 15766 2993 164132 94555 1 BAYLOR COLLEGE OF MEDICINE PO BOX 301207 DALLAS TX 70690 741613878 501(C)(3) 18750 0 RESEARCH GRANT BRIGHAM & WOMEN'S HOSPITAL POB 3149 BOSTON MA 02241 042312909 501(C)(3) 56250 0 RESEARCH GRANT UNIVERSITY OF VIRGINIA 1001 N EMMET ST CHARLOTTESVILLE VA 22903 546001796 501(C)(3) 18750 0 RESEARCH GRANT NATIONAL ORGANIZATION FOR RARE DISORDERS DEPT 5430 PO BOX 4110 WOBURN MA 01888 133223946 501(C)(3) 125000 0 TSC TRAVEL AND LODGING ASSISTANCE PROGRAM UNIVERSITY OF CALIFORNIA 10889 WILSHIRE BLVD SUITE 700 LOS ANGELES CA 90095 956006143 501(C)(3) 20000 0 RESEARCH GRANT COLUMBIA UNIVERSITY IN THE CITY OF NEW YORK 615 W 131ST STREET 6TH FLOOR MAIL CODE 8725 NEW YORK NY 10027 135598093 501(C)(3) 75000 0 RESEARCH GRANT UNIVERSITY OF CALIFORNIA IRVINE 228 ALDRICH HALL IRVINE CA 92697 952226406 501(C)(3) 18750 0 RESEARCH GRANT UNIVERSITY OF MARYLAND BALTIMORE PO BOX 41428 BALTIMORE MD 21203 526002033 501(C)(3) 56250 0 RESEARCH GRANT UNIVERSITY OF TEXAS SOUTHWESTERN MEDICAL CENTER 5323 HARRY HINES BLVD DALLAS TX 75390 756002868 501(C)(3) 56250 0 RESEARCH GRANT UNIVERSITY OF TEXAS DALLAS 800 WEST CAMPBEL ROAD SP2 27 RICHARDSON TX 75080 751305566 501(C)(3) 18750 0 RESEARCH GRANT UNIVERSITY OF ALABAMA BIRMINGHAM 1720 2ND AVE SOUTH AB990 BIRMINGHAM AL 32294 636005396 501(C)(3) 91649 0 RESEARCH GRANT UNIVERSITY OF NEW ENGLAND 11 HILLS BEACH RD ROOM 207 BIDDEFORD ME 04005 010211810 501(C)(3) 20000 0 RESEARCH GRANT BOSTON CHILDREN'S HOSPITAL PO BOX 414413 BOSTON MA 02241 042774441 501(C)(3) 57409 0 RESEARCH GRANT 13 0 PART I, LINE 2: THE TSC ALLIANCE HAS FUNDED $37 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 MERITS, 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 STEVEN ROBERDS CHIEF SCIENTIFIC OFFICER 214711 0 22497 0 0 0 7291 0 19272 0 263771 0 0 0 KARI LUTHER ROSBECK PRESIDENT & CEO 201924 2040 21822 220 0 0 7012 0 22405 0 253163 2260 0 0 DEAN RAGER-AGUIAR VICE PRESIDENT, TRANSLATIONAL RESEAR 159369 0 18356 0 0 0 5660 0 22431 0 205816 0 0 0 CYNTHIA ARCURI CHIEF FINANCIAL OFFICER 161389 8494 15600 821 0 0 5588 0 3894 0 186471 9315 0 0 PART I, LINE 5 KARI LUTHER ROSBECK, STEVE ROBERDS, CYNTHIA ARCURI, DEAN AGUIAR, JAYE ISHAM, LISA MOSS, AND ASHLEY POUNDERS ALL HAVE INCENTIVE COMPENSATION EQUAL TO A PERCENTAGE OF THEIR SALARIES BASED ON KEY PERFORMANCE OBJECTIVES AS ESTABLISHED BY THEIR COMPENSATION COMMITTEE. PART I, LINE 6 KARI LUTHER ROSBECK, STEVE ROBERDS, CYNTHIA ARCURI, DEAN AGUIAR, JAYE ISHAM, LISA MOSS, AND ASHLEY POUNDERS ALL HAVE INCENTIVE COMPENSATION EQUAL TO A PERCENTAGE OF THEIR SALARIES BASED ON KEY PERFORMANCE OBJECTIVES AS ESTABLISHED BY THEIR COMPENSATION COMMITTEE. FORM 990, PART III, LINE 4A, DESCRIPTION OF PROGRAM SERVICE: THE PRECLINICAL CONSORTIUM INVITES COMPOUND NOMINATIONS FROM ACADEMIC AND INDUSTRY RESEARCHERS 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. A MEMBERS MEETING IS HELD ANNUALLY 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. IN 2023, TWO NEW INDUSTRY MEMBERS JOINED THE CONSORTIUM WITH A TOTAL OF 9 ACTIVE INDUSTRY MEMBERS AT THE END OF THE YEAR. THE CONSORTIUM HAS HAD 22 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. THE CONSORTIUM TESTED 17 UNIQUE COMPOUNDS IN 2023, RAISING THE TOTAL TESTED TO 82 SINCE 2016. MANY COMPOUNDS WILL ENTER PRECLINICAL TESTING, THOUGH ONLY SOME WILL ADVANCE TO CLINICAL TESTING DUE TO LACK OF EFFICACY AND SAFETY. 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 $40 MILLION BY THE NATIONAL INSTITUTES OF HEALTH (NIH) AND FOOD AND DRUG ADMINISTRATION (FDA) 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 TSCWAS THE FIRST PREVENTATIVE TRIAL IN THE UNITED STATES FOR ANY FORM OF EPILEPSY. INITIAL RESULTS FROM THE STUDY WERE REPORTED THIS YEAR. IN THE PREVENT TRIAL, INFANTS WITH TSC RECEIVED REGULAR EEG MONITORING AND THOSE WHO DEVELOPED EEG ABNORMALITIES WERE PLACED ON EITHER VIGABATRIN (SUPPLIED BY LUNDBECK) OR A PLACEBO. ANY INFANT WHO SUBSEQUENTLY DEVELOPED CLINICAL SEIZURES WAS IMMEDIATELY PLACED ON STANDARD TREATMENT. RESULTS OF THE STUDY SHOWED THAT PREVENTATIVE TREATMENT WITH VIGABATRIN BEGINNING WHEN EEG BIOMARKER CHANGES WERE OBSERVED DELAYED THE ONSET AND LOWERED THE OVERALL OCCURRENCE OF INFANTILE SPASMS IN INFANTS WITH TSC. HOWEVER, THERE WAS NO SIGNIFICANT DIFFERENCE BETWEEN THE TREATMENT GROUP AND THE PLACEBO GROUP IN TERMS OF DRUG-RESISTANT EPILEPSY, FOCAL SEIZURES, OR COGNITIVE AND BEHAVIORAL OUTCOMES AT TWO YEARS OF AGE. IN THIS TRIAL, THE BIGGEST PREDICTOR OF POORER DEVELOPMENTAL OUTCOMES WAS THE AGE AT WHICH THE EEG BIOMARKER CHANGES OCCURRED: THOSE INFANTS WHOSE EEG CHANGES OCCURRED PRIOR TO SEVEN MONTHS OF AGE HAD, ON AVERAGE, LOWER COGNITIVE AND BEHAVIORAL SCORES THAN THOSE WHOSE EEG CHANGES OCCURRED LATER, REGARDLESS OF TREATMENT VIA VIGABATRIN VERSUS PLACEBO. THIS SUGGESTS THAT RAPID DIAGNOSIS OF TSC IN INFANTS, PRIOR TO HAVING ANY SEIZURES, FOLLOWED BY A CLOSE MONITORING AND RAPID INITIATION OF TREATMENT WHEN SEIZURES BEGIN IS CRITICAL FOR ENSURING THE BEST OUTCOMES POSSIBLE. 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 BEFORE THE ONSET OF SEIZURES TO TEST THE EFFECTS OF SIROLIMUS ON PREVENTING OR DELAYING SEIZURE ONSET. THE STOPPING TSC ONSET AND PROGRESSION 2 (STOP-2) TRIAL ENROLLED FIVE INFANTS AT CINCINNATI CHILDREN'S HOSPITAL MEDICAL CENTER AS AN INITIAL SAFETY STUDY. THE SECOND STAGE OF THE TRIAL IS NOW CALLED "SIROLIMUS TSC EPILEPSY PREVENTION STUDY (TSC-STEPS) AND THE TSC ALLIANCE HAS CONTRIBUTED $200,000 TO ENABLE THE TRIAL TO BE EXPANDED TO ADDITIONAL SITES ACROSS THE COUNTRY. THE 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 IN 2019 FOR A SECOND 5-YEAR FUNDING PERIOD. THE TSC ALLIANCE FUNDS A PORTION OF CLINICAL RESEARCH COORDINATORS' SALARIES AT 5 SITES. IN 2021, THE TSC ALLIANCE AWARDED A TWO-YEAR CLINICAL RESEARCH TSC-DSC FELLOWSHIP TO FELIX CHAN, PHD, AT BROWN UNIVERSITY WHO MOVED IN 2022 TO ASTON UNIVERSITY IN BIRMINGHAM, ENGLAND. FORM 990, PART III, LINE 4B, DESCRIPTION OF PROGRAM SERVICE: SUPPORT SERVICES IS DEDICATED TO SUPPORTING INDIVIDUALS AND FAMILIES AFFECTED BY TSC THROUGH OUTREACH PROGRAMS, SUPPORT SERVICES, IMPROVED MEDICAL ACCESS, AND EDUCATIONAL RESOURCES. IN 2023, THE TSC ALLIANCE IMPLEMENTED A NEW COMMUNITY SUPPORT MODEL THAT PUTS THE TSC COMMUNITY CENTER FOCUSING ON LOCAL RESOURCES, SUPPORT SERVICES, AND ACCESS TO MEDICAL CARE IN CONJUNCTION WITH ENERGIZING THE CURRENT AND FUTURE VOLUNTEER LEADERS TO PROPEL THE TSC ALLIANCE TO THE NEXT LEVEL. THIS MODEL UNITED 36 COMMUNITY ALLIANCES AND ORGANIZED THEM INTO 15 COMMUNITY REGIONS, LED BY MORE THAN 150 DEDICATED LEADERS THROUGHOUT THE UNITED STATES. THE COMMUNITY PROGRAMS TEAM INVESTED MORE THAN 900 TRAINING HOURS TO OUR LEADERS, TO ENSURE THEY WERE WELL EQUIPPED TO SUPPORT AND EMPOWER THE TSC COMMUNITY. AS A RESULT, WE PROVIDED 4,278 PEER-TO-PEER SUPPORT ENGAGEMENTS AND FACILITATED 50 SCHOOL MEETINGS, WHICH INVOLVED TSC 101 TRAINING AND IEP CONSULTATIONS. IN ADDITION, TWO "ASK AN ADVOCATE" WEBINARS ATTRACTED 94 REGISTRANTS. THE COMMUNITY PROGRAMS TEAM COLLABORATED WITH KEY STAFF TO HOST SEVEN RESEARCH AND CORPORATE PARTNER WEBINARS. THESE WEBINARS ATTRACTED 312 LIVE ATTENDEES AND GARNERED 832 RECORDED VIEWS, WITH 801 LANDING PAGE VIEWS AS OF DECEMBER 31, 2023. THE TSC ALLIANCE ALSO PARTNERED WITH THE LAM FOUNDATION TO CO-HOST FOUR EDUCATIONAL CONFERENCE SERIES HELD IN SEATTLE, WA; DENVER, CO; BIRMINGHAM, AL; WASHINGTON, DC. THESE CONFERENCES ATTRACTED 350-PLUS PARTICIPANTS. THE TSC ALLIANCE'S TSC NAVIGATOR IS AN EASY-TO-USE, INTERACTIVE ONLINE TOOL TO HELP GUIDE INDIVIDUALS AND FAMILIES THROUGH THE COMPLEXITIES OF TSC ACROSS THE LIFESPAN, PROACTIVELY MANAGE THEIR CARE, AND LIVE THEIR FULLEST LIVES. USERS CAN ACCESS INFORMATION BASED ON THE AGE OF ONE'S DIAGNOSIS, SUCH AS PRENATAL, CHILDHOOD, OR ADULT, TO HELP DETERMINE WHICH STEPS WILL HELP EMPOWER THEM THROUGHOUT THEIR INDIVIDUAL JOURNEYS. IN 2023, THE TOOL HAD 2,379 USERS. EIGHT INDIVIDUALS OR FAMILIES WERE ALSO SUPPORTED FOR MEDICATION ACCESS ISSUES VIA OUR TSC NAVIGATOR PROGRAM DURING 2023, DOWN FROM 55 IN 2022, CONSISTENT WITH THE GOAL OF PROACTIVELY HELPING PEOPLE AVOID MEDICATION ACCESS ISSUES. FORM 990, PART III, LINE 4C, DESCRIPTION OF PROGRAM SERVICE: PUBLIC HEALTH EDUCATION INCREASES AWARENESS OF TSC THROUGHOUT THE GENERAL PUBLIC TO BROADEN THE SCOPE OF SUPPORT AND UNDERSTANDING BEYOND TSC INDIVIDUALS AND THEIR FAMILIES; THESE EFFORTS ALSO WORK TO HELP REACH THE UNDIAGNOSED AND NEWLY DIAGNOSED. DURING 2023, THE TSC ALLIANCE PRODUCED TWO ISSUES OF ITS NATIONAL MAGAZINE, PERSPECTIVE, WHICH IS MAILED TO APPROXIMATELY 18,500 CONSTITUENTS AS WELL AS POSTED ON THE WEBSITE. THE TSC ALLIANCE'S PODCAST SERIES, CALLED TSC NOW, PRODUCED EIGHT EPISODES WITH 2,180 TOTAL LISTENS. FIVE ISSUES OF THE ELECTRONIC TSC MATTERS NEWSLETTER WERE DISTRIBUTED TO 17,514 RECIPIENTS. THE TSC ALLIANCE'S WEBSITE INCREASES AWARENESS AND PROVIDES EXTENSIVE EDUCATION THROUGH A MONTHLY AVERAGE OF 26,916 PAGE VIEWS. THE TSC ALLIANCE ALSO RELIES HEAVILY ON SOCIAL MEDIA TO EDUCATE CONSTITUENTS AND PROMOTE NEW RESOURCES AND EVENTS. ITS PRIVATE FACEBOOK TSC DISCUSSION GROUP BOASTS MORE THAN 10,800 MEMBERS FROM AROUND THE WORLD, WHILE ITS TWITTER ACCOUNT HAS 2,684 FOLLOWERS AND INSTAGRAM HAS 3,432 FOLLOWERS. TO INCREASE PUBLIC AWARENESS, THE TSC ALLIANCE PARTICIPATED IN THE 11TH ANNUAL TSC GLOBAL AWARENESS DAY ON MAY 15 AS WELL AS TSC AWARENESS MONTH THROUGHOUT MAY. THE TSC ALLIANCE ALSO HEAVILY PROMOTED INFANTILE SPASMS AWARENESS WEEK (DECEMBER 1 TO 7). THESE AWARENESS CAMPAIGNS ALONG WITH THE ORGANIZATION'S VARIOUS NEWS RELEASES AND SOCIAL MEDIA OUTREACH EFFORTS CULMINATED IN MORE THAN 812 MILLION CUMULATIVE IMPRESSIONS AND ENGAGEMENTS. FORM 990, PART VI, SECTION A, LINE 6 MEMBERSHIP IN THE CORPORATION 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 IS 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 ALLOWS INDIVIDUALS TO STATE THEIR INTENT TO BE A MEMBER FOR THE PURPOSE OF GOVERNANCE. THERE WERE NO LEVELS TO MEMBERSHIP IN 2021. 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 2023: BOARD MEMBER MUSTAFA SAHIN, PH.D., M.D., IS EMPLOYED AT BOSTON CHILDREN'S HOSPITAL, WHICH RECEIVED $57,409 IN GRANTS AND $6,300 IN FEES FOR SERVICES 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 $3,420 IN FEES FOR SERVICES FOR PARTICIPATION IN THE TSC NATURAL HISTORY DATABASE. BOARD MEMBER JESSICA KREFTING, RN, IS EMPLOYED AT UNIVERSITY OF ALABAMA AT BIRMINGHAM, WHICH RECEIVED $91,649 IN GRANTS AND $9,010 IN FEES FOR SERVICES FOR PARTICIPATION IN THE TSC NATURAL HISTORY DATABASE. BOARD MEMBER AND IMMEDIATE PAST CHAIR PETER CRINO, MD, PHD, IS CHAIR OF THE DEPARTMENT OF NEUROLOGY AT THE UNIVERSITY OF MD, WHICH RECIEVED $56,250 OF GRANT PAYMENTS." FORM 990, PART VI, SECTION B, LINE 15 "THE COMPENSATION COMMITTEE REVIEWS AND APPROVES THE SALARIES OF THE PRESIDENT/CEO, CHIEF SCIENTIFIC OFFICER, 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. FORM 990, PART XII, LINE 2C: THIS PROCESS HAS NOT CHANGED FROM THE PRIOR YEAR. FORM 990 AMENDED RETURN THE FORM 990 WAS AMENDED TO INCLUDE THE JOINT COST ALLOCATION THAT WAS INADVERTENTLY LEFT OFF OF PART IX LINE 26. TSC ALLIANCE ENDOWMENT FUND INC 8737 COLESVILLE ROAD NO 400 SILVER SPRING MD 20910 521926919 SUPPORT THE MISSION OF THE TSC ALLIANCE 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 572625 ACTUAL CASH RECEIVED