activities · 8
what they do
direct service · 4
- Community memorial and awareness exhibits Patient and Family Storytelling ProgramsDisplays artwork created in memory of children lost to Tay-Sachs disease, including pieces by Ralph Keefner, and shares photographs of affected children submitted by their families to raise awareness and honor lives impacted by the disease.
- Community memorial and awareness exhibits Patient and Family Storytelling ProgramsDisplays artwork and photographs honoring children affected by Tay-Sachs disease, including pieces created in memory of affected family members and submissions from parents.
- Fundraising to support Tay-Sachs disease research Disease-Specific Research FundraisingRaises funds to support global research initiatives for Tay-Sachs disease, having raised over $7.6 million since 2007.
- Fundraising to support research initiatives Disease-Specific Research FundraisingHas raised over $7.6 million since 2007 to fund global research efforts targeting Tay-Sachs disease.
research · 4
- Funding and support for global Tay-Sachs research initiativesSupports clinical trials and funds global research efforts, having funded 25 research projects worldwide to advance medical knowledge and treatment development for Tay-Sachs disease.
- Gene therapy and clinical research for Tay-Sachs and Sandhoff diseases Genomic and Translational Biomedical ResearchConducts and funds gene therapy research and supports clinical trials aimed at developing treatments for Tay-Sachs and Sandhoff diseases, with ongoing scientific progress reported as of 2026.
- Gene therapy and clinical research for Tay-Sachs and Sandhoff diseasesConducts and funds gene therapy research for Tay-Sachs and Sandhoff diseases, with active development of potential clinical trials and reported scientific progress as of 2026. The foundation has funded 25 global research efforts and continues to advance medical knowledge in pursuit of viable treatments.
- Gene therapy research for Tay-Sachs and Sandhoff diseases Genomic and Translational Biomedical ResearchConducts and advances gene therapy research for Tay-Sachs and Sandhoff diseases, with ongoing work toward potential clinical trials and reported scientific progress as of 2026.
strategies · 15
how they think
Theories of action extracted from this org's own source material. Click any to see the full field of orgs running the same approach.
- Advocacy and education to increase visibility and prevention rare_disease_advocacyThrough rare disease advocacy and public education, the foundation aims to raise awareness, influence policy, promote carrier screening, and encourage prenatal testing to prevent new cases in high-risk populations.
- Engaging families as partners in research and remembrance family-engaged_researchThe foundation involves affected families not only as collaborators in accelerating research but also through personal, artistic expression to honor affected children, strengthening community engagement and research motivation.
- Enzyme-targeted therapy development enzyme_targeted_therapyThe organization focuses on developing therapies for Tay-Sachs disease by targeting the insufficient Hex-A enzyme activity and the resulting accumulation of GM2 waste in brain and nerve cells. This includes strategies to increase Hex-A enzyme production, reduce GM2 accumulation, or reduce GM2 production.
- Family-Centered Remembrance through Artistic Expression family-centered remembranceThe organization honors children impacted by Tay-Sachs disease by facilitating personal artistic expression from affected families, providing a means of remembrance and coping.
- Family-Engaged Research family-engaged_researchThe organization actively involves families affected by Tay-Sachs disease as partners in research, believing that their engagement is crucial for driving scientific progress.
- Family-engaged research family_engaged_researchThe organization actively involves families affected by Tay-Sachs disease as partners in research efforts, believing that their engagement drives scientific progress.
- Gene Therapy using Viral Vectors gene therapy using viral vectorsThe organization utilizes viral vectors to deliver functional Hex-A genes into brain cells, thereby enabling the production of the necessary enzyme to combat Tay-Sachs disease.
- Gene therapy using viral vectors gene_therapy_viral_vectorsThe organization utilizes viral vectors in gene therapy to deliver functional Hex-A genes directly to brain cells, thereby enabling the production of the necessary enzyme.
- Preventive Genetic Testing and Carrier Screening preventive_genetic_testingGiven the lack of a cure, the organization emphasizes genetic testing for Tay-Sachs carriers and prenatal testing, especially for high-risk couples and populations, as a primary preventive measure.
- Rare Disease Advocacy rare_disease_advocacyThe organization employs advocacy to raise the profile of Tay-Sachs disease, aiming to accelerate research, influence policy decisions, and foster community support for those affected.
- Rare disease advocacy rare_disease_advocacyThe organization employs advocacy to increase the visibility of Tay-Sachs disease, which in turn accelerates research, influences policy, and builds community support for the cause.
- Stem Cell-Based Enzyme Production stem cell-based enzyme productionThe organization explores the use of autologous stem cell treatment as a method to produce the Hex-A enzyme, with the goal of reducing complications associated with other therapeutic approaches.
- Stem cell-based enzyme production stem_cell_therapyThe organization explores the use of autologous stem cell treatment as a method to produce the Hex-A enzyme, aiming to reduce complications associated with other therapeutic approaches.
- Targeting Hex-A Enzyme Deficiency for Tay-Sachs Treatment enzyme-targeted therapy developmentThe organization focuses on developing therapies that address the insufficient Hex-A enzyme activity characteristic of Tay-Sachs disease, aiming to either increase enzyme production, reduce GM2 accumulation, or decrease GM2 production to mitigate the disease's effects.
- Targeting Hex-A enzyme deficiency to treat Tay-Sachs disease enzyme-targeted therapy developmentThe foundation focuses on therapeutic strategies that address the root cause of Tay-Sachs—insufficient Hex-A enzyme activity—by increasing enzyme production, reducing GM2 accumulation, or enabling functional enzyme expression through gene or stem cell therapy.
named programs · 3
what they call their work
Advocacy and Awareness
Drives awareness, policy, and hope for families affected by Tay-Sachs disease through advocacy efforts and community voices.
Research Funding
Funds global research efforts focused on overcoming GM2 accumulation, increasing Hex-A production, and exploring potential treatments like chaperone drugs, inhibitor substrates, enzyme replacement therapy, gene therapy, and stem cell treatment for Tay-Sachs disease.
Tay-Sachs Clinical Trials Information
Provides information and guidance for families navigating research options and clinical trials for Tay-Sachs disease.
relationships · 5
who they work with
- CRISPR Partner — Exploring CRISPR technology for gene correction in Tay-Sachs disease research.
- Constant Contact Partner — Emails are serviced by Constant Contact.
- National Tay-Sachs & Allied Diseases Association Partner — Collaborates by referencing NTSAD's list of certified screening locations for carrier testing.
- National Tay-Sachs & Allied Diseases Association Partner — References NTSAD for a listing of certified carrier screening locations and provides their contact information for more information about Tay-Sachs disease and carrier testing.
- Researchers Partner — Families and the foundation partner with researchers to advance Tay-Sachs disease research.