Introduction
The TANGO2 Research Foundation is a patient-led nonprofit organization that funds and coordinates scientific research, supports affected families, promotes early diagnosis, and works toward effective treatments and a cure for TANGO2 Deficiency Disorder.
History and Background
TANGO2 Deficiency Disorder, commonly known as TDD, was first identified as a distinct human genetic disease in 2016 following advances in genomic technologies such as whole-exome sequencing.
At that time, few people had been diagnosed, and researchers possessed limited information about how changes in the TANGO2 gene caused neurological, metabolic, developmental, and cardiac complications.
The Foundation’s history is closely connected to Mike and Kasha Morris and their son, Ryan. After Ryan began experiencing developmental regression and other symptoms shortly after his first birthday, the family spent approximately 15 years seeking a diagnosis. Genetic testing finally identified TANGO2 Deficiency Disorder in 2017.
After connecting with other affected families through an online community, Mike and Kasha Morris began developing an organization that could unite families, researchers, clinicians, and advocates. The first TANGO2 Research Foundation board meeting was held in 2018.
The Foundation is registered as a nonprofit corporation in Connecticut and is exempt from United States federal income tax under Section 501(c)(3) of the Internal Revenue Code.
Mission and Purpose
The TANGO2 Research Foundation’s mission is to lead the way in finding a cure for TANGO2 Deficiency Disorder.
It pursues this mission by funding, coordinating, and guiding scientific research that improves understanding of the cellular, biochemical, metabolic, neurological, and cardiac effects of TANGO2 gene variants.
Its strategic priorities are to accelerate research and therapy discovery, advocate for the TANGO2 community, promote awareness, support and connect families, and improve the accuracy and speed of diagnosis.
The Foundation also works to improve clinical care and quality of life while research toward effective treatments and a cure continues.
Governance and Structure
The Foundation is governed by an international board of directors comprising parents, clinicians, researchers, and advocates. The board establishes strategic priorities, oversees organizational resources, and guides investments in research, education, family support, and scientific collaboration.
Mike Morris serves as president, while Kasha Morris serves as secretary. Both are co-founders of the Foundation. The board also includes a vice president, treasurer, medical professionals, scientific researchers, parents, and community representatives.
Board positions are voluntary and uncompensated. The Foundation requires all serving directors to contribute financially through personal gifts or support generated through their families, businesses, and professional networks.
The executive director oversees the Foundation’s daily operations, programs, fundraising, stakeholder engagement, financial management, and organizational strategy.
An international Scientific Advisory Board provides independent advice on research priorities and funding decisions. Its members include specialists in genetics, cardiology, neurology, metabolic medicine, molecular biology, and rare-disease research.
The organization also operates executive, research, fundraising, outreach, and early detection and diagnosis committees. These groups involve board members, professionals, volunteers, researchers, and family representatives.
Funding and Grants
The TANGO2 Research Foundation finances its work through individual contributions, recurring monthly donations, community fundraising campaigns, corporate partnerships, philanthropic grants, merchandise purchases, memorial funds, and support from affiliated organizations.
Its research grant program provides seed and pilot funding for rigorous studies examining TANGO2 Deficiency Disorder. These awards allow researchers to test new concepts, evaluate feasibility, refine methodologies, and generate evidence that may support larger studies.
The Foundation has awarded approximately US$1.38 million in research funding since its establishment. Its portfolio supports basic science, clinical research, natural-history studies, therapeutic exploration, patient-centred outcomes, and tools that may improve daily life for people with TDD.
The TDD Research Pathways Grant supports early-career researchers, undergraduate and graduate students, postdoctoral fellows, medical residents, and clinical or research fellows. It provides up to US$15,000 for one-year pilot studies and innovative research projects.
Applicants from any country may apply for the Research Pathways Grant, subject to its eligibility requirements. The program emphasizes mentorship, institutional support, collaboration, professional development, translational research, and improved outcomes for people living with TDD.
The Foundation also offers scientific travel grants of up to US$1,000 to help researchers present original TANGO2 research at medical meetings, conferences, and symposia.
Major Programs and Initiatives
The research funding portfolio supports investigators at different career stages and institutions around the world. Funded studies address disease mechanisms, neurological and cardiac complications, metabolism, biomarkers, natural history, clinical care, and potential treatments.
The TANGO2 Natural History Study collects clinical and molecular information from individuals with TDD. Its objectives include developing a clinical database and biorepository, improving knowledge of disease progression, supporting patient identification, and providing resources for future therapeutic research.
The patient registry helps researchers and the Foundation understand the size, location, characteristics, and needs of the international TANGO2 community. It can also support study recruitment and preparation for future clinical research.
Family conferences and research symposia bring together patients, caregivers, clinicians, researchers, and advocates. These events enable families to learn about scientific progress while helping researchers understand the priorities and lived experiences of affected people.
The Research Learning Network strengthens communication and collaboration among investigators, clinicians, families, and other stakeholders engaged in TANGO2 research.
The Foundation also provides family resources, regional support, clinical-care information, emergency protocols, educational videos, healthcare professional resources, and access to the TDD Clinician Hotline.
TANGO2 PubNav is an AI-assisted literature exploration and community platform that consolidates TDD research information and helps families, researchers, and clinicians navigate relevant scientific publications.
Impact and Examples of Work Funded
The Foundation reports approximately US$1.38 million invested across 33 research projects involving 38 funded researchers, 27 institutions, and nine countries.
Its research roadmap prioritizes the management of life-threatening symptoms, improved independence and quality of life, prevention of functional decline, cellular responses to medicines, and clinical studies examining ways to prevent metabolic crises and rhabdomyolysis.
One funded project is developing the TANGO2 Natural History Study and associated biorepository at Baylor College of Medicine. The Foundation awarded US$85,000 to support clinical information collection, molecular investigations, patient recruitment, and resources for future treatments.
Another project received US$20,000 in joint support from the Foundation and Uplifting Athletes to develop and study human neurons derived from induced pluripotent stem cells. The research seeks to identify neurological pathways associated with TDD and inform potential treatment strategies.
A further jointly funded US$20,000 study used zebrafish models to investigate changes in lipid metabolism associated with disease onset and progression. Its findings are intended to improve understanding of disease mechanisms and possible treatment pathways.
The Foundation has enrolled more than 90 patients in its Natural History Study, produced more than 50 educational videos, supported over 25 TANGO2-related research articles and publications, and organized family conferences and a research symposium.
Its work has helped transform TANGO2 Deficiency Disorder from a newly identified and poorly understood condition into a growing area of coordinated international scientific research.
Conclusion
The TANGO2 Research Foundation combines patient leadership, targeted research funding, scientific guidance, international collaboration, clinical resources, and family support to accelerate progress against an extremely rare genetic disorder. By connecting families with researchers and clinicians, the Foundation is building the knowledge, evidence, and research infrastructure required to improve diagnosis, strengthen care, develop treatments, and ultimately find a cure.
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