National Tuberous Sclerosis Association Inc

Goal 1: Accelerate research.
We will accelerate the progress in TSC research through federal funding, collaborative research and resource tools.

Goal 2: Improve access and quality of care.
We will support quality TSC care and empower consumers in diverse communities to access it.

Goal 3: Support and empower consumers.
We will provide support and educational services to individuals/caregivers throughout the lifespan to improve quality of life around the world.

Goal 4: Educate and mobilize to increase investment
We will educate the public about those affected by TSC and the benefits of increased investment in TSC research for related disorders such as Autism, Epilepsy, and LAM, and mobilize them to join and support our programs in order to increase annual revenue from both philanthropic and non-philanthropic sources.

Goal 5: Build and strengthen organization
We will build organizational unity and capacity to strengthen the economic viability and growth of the TSC Alliance in order to achieve our mission.

Goal 1: Accelerate research.
Enable researchers to access and utilize the wide variety of clinical, omics and Patient Reported Outcomes (PRO) data collected by or shared with TSC Alliance
Increase the number and diversity of individuals with TSC participating in research
Expand quantitative clinical data collected in the Natural History Database (NHD) / Biosample Repository (BSR).
Develop prognostic, predictive and stratification biomarkers
Drive development of quantitative patient-reported outcome measures for impactful aspects of TSC
Cultivate and engage industry partners to use the Preclinical and Clinical Research Consortia
Enhance collaboration with academic researchers by raising funds to pay for preclinical testing of nominated compounds or other technologies
Transfer TSC Clinical Research Consortium to the TSC Alliance and establish membership model for industry participation and revenue generation
Advocate for federal and state funding for research
Expand racial and ethnic diversity of research grant applicants
Demonstrate impact of TSC Alliance funding

Goal 2: Improve Access and Quality of Care.
Expand healthcare access
Improve quality and comprehensiveness of healthcare
Build evidence-based to support updates to best practice guidelines for TSC clinical care

Goal 3: Support and empower consumers.
Strengthen our volunteer leadership model to address gaps in resources, support, advocacy and medical access and train our leaders to represent a more inclusive and collaborative community
Establish a stronger collaboration with TSC Clinics, healthcare providers and leaders to further support and provide resources to TSC families
Improve TSC community support and resources through cultivating and growing ties with new and current partners
Grow global expertise as a way to better support the TSC community

Goal 4: Educate and mobilize to increase investment
Launch public phase and complete capital campaign to enable growth of research platform through partnerships with corporations, foundations and individuals
Educate the community and other donors about the role and opportunities associated with Endowment.
Increase annual unrestricted revenue
Broadcast TSC information, TSC community stories and research accomplishments to increase awareness and position the TSC Alliance as a leader in the rare disease space
Create targeted strategies to better reach underserved TSC communities and educate key medical providers

Goal 5: Build and strengthen organization
Adhere to financial management best practices while maintaining appropriate infrastructure for organizational growth
Maintain database of current, reliable information of individuals with TSC, their families and supporters complying with GDPR
Grow and maintain board of directors reflecting the diversity of our constituency and with skill sets needed to realize the strategic plan
Invest in professional development to cultivate the next generation of staff team leaders

With a professional staff of 23, the TSC Alliance is governed by a 27-member Board of Directors as of January 1, 2024, comprised of people affected directly by the disease, such as parents or adults with TSC, as well as business professionals, researchers and clinicians. The staff and Board have achieved 100% giving to demonstrate their leadership in the fight against TSC. Since 2010, the organization has placed strategic focus on achieving recognition by industry watchdog organizations, earning a 4 Star Rating from Charity Navigator, the Candid (Guidestar) Exchange Platinum Seal, accreditation by the Better Business Bureau Wise Giving Program and being named as a Top Nonprofit from GreatNonprofits from 2012-2023. The TSC Alliance is headquartered in Silver Spring, Maryland with field staff in CA, DE, LA, MA, MI, MS, NM, PA, TN.

The staff and Board are also supported by an army of more than 2.200 volunteers. Working together, the TSC Alliance strives to improve the quality of life for every individual and family affected by TSC by focusing on three main pillars: Research, Advocacy and Outreach. Each pillar plays a pivotal role in our mission.

Research: The TSC Alliance 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. In 2011, we launched the Unlock the Cure Campaign with the goal to move potential treatments along the drug discovery path more rapidly. As a result, we have tested 83 candidate drugs in the Preclinical Consortium, enrolled nearly 2,800 individuals in the Natural History Database linked with 2,700 samples in the Biosample Repository, and have engaged in two clinical biomarker studies, a rare diseases clinical research network and the first and second preventative clinical trial for epilepsy in the United States in TSC.

Advocacy: For more than 20 years, the TSC Alliance, partners and grassroots volunteers have worked tirelessly to create a vibrant government advocacy program with the goal of making sure the Federal government continues to focus time, energy and dollars on TSC research. These efforts have translated into more than $505 million in Federal funding for TSC research since 2002 through the National Institutes of Health and appropriations to the Department of Defense’s Congressionally Directed Medical Research program.

Outreach: Our community outreach efforts are designed to offer support and provide education. We employ a full-time advocate, offer a robust website, publish a national magazine, produce targeted brochures on TSC manifestations, and host a multi-channel digital platform. Additionally, we have 14 regional volunteer branches covering all 50 states and six global alliances, increasing our worldwide footprint.

• Accelerate research: spent $14.1 million into research 2019-June 30, 2024; $121 million appropriated for TSCRP since FY02; 83 mechanisms or combinations tested by Preclinical Consortium with 23 companies; 3 companies moving into clinical trials; 2811 biosamples, $2,659,023 (2019-2024) awarded in research grants; International TSC Research Conference held in 2019, 2021 and 2023, and Newborn Screening Innovation Workshop in 2020, TAND Workshop in 2022 and Reproductive Perinatal Health Workshop in 2024.

• Improve access and quality of care: state advocacy initiatives with secured appropriations of $1.5 million in Missouri and $2.75 million in Maryland; ongoing relationships with 19 industry partners and four specialty pharmacy partners; TSC Focus Group established and Board approved new standards for TSC Clinic and Centers of Excellence; 200 child neurologists participated in the Harmful and Disruptive Behaviors CME Symposium at CNS, developed by the Child Neurology Foundation with the TSC Alliance as lead advocacy partner and assisting in curriculum development; secured funding for a CME with 2,151 participants; 50% TSC designated clinics see individuals of all ages.

* Support and empower constituents: 4,278 peer to peer support contacts in 2023; 904 volunteer training hours 2023; 50 school meetings in 2023; built and maintained working partnership with 17 national organizations/collaborations; 7 webinars attended virtual meetings in 2023 with 312 live attendees and 832 recorded views. 63% of new March on Capitol Hill attendees took TSC Academy in preparation for advocacy on Capitol Hill. Maintained 6 global partnerships with 3 new global TSC Clinics, bringing the total 10.

*Educate and mobilize to increase investment: raised $16,871,412 for research ; Endowment Fund assets $5,921,817 as of 12/31/23; $2,421,439 net was raised through special events in 2019, $1,227,362 in 2020, $1,143,605 in 2021; $1,443,671 in 2022; $1,284,007 in 2023; 8,283 individuals donated online to crowdfunded events in 2023; in 2023 garnered 812 million impressions; marketing/communications presented findings/recommendations early 2020 and organization moved forward with planning and decisions for name change/rebranding in 2021, formally beginning May 15.

• Build and sustain organization: 4-star CN rating, BBB Giving accreditation, Platinum Candid status, GreatNonprofit in 2023; exp ratios 79.86% program/20.14% admin and fundraising for FY23; staff attended 26 professional conferences in 2023; added 3 new board members; Science and Medical Team to 6 FTEs.