The TANGO2 Innovation Fund celebrates the extraordinary clinical and scientific professionals who are advancing research and providing compassionate care for those affected by TANGO2 deficiency disorder.
The TANGO2 Innovation Fund Campaign funds research initiatives that drive the latest breakthroughs and treatments for TANGO2 deficiency disorder (TDD). As the only organization dedicated exclusively to finding a cure, we are proud to share researchers’ stories and highlight the meaningful impact of their work.
Voices of Innovation
For over five years, these dedicated clinicians and researchers have partnered with the TANGO2 Research Foundation and the broader TDD community, pushing the boundaries of knowledge, care, and hope for better treatments, health outcomes and a cure. Click the play button to hear their voices and learn more.
Lina Ghaloul-Gonzalez, MD
Dr. Ghaloul-Gonzalez, a geneticist at UPMC Children’s Hospital of Pittsburgh, cares for TANGO2 warriors and their families in the clinic. She is also an Assistant Professor of Pediatrics in the Division of Genetic and Genomic Medicine, with a secondary appointment in the Department of Human Genetics at the University of Pittsburgh Graduate School of Public Health. A longtime partner of the foundation, she has received multiple research grants, serves on the T2RF Research Committee, and has been a key contributor to the foundation’s PCORI-funded initiatives, helping to advance care and understanding for TANGO2 warriors worldwide.
Seema Lalani, MD
Dr. Lalani credited with first characterizing TDD in 2016, is a Professor of Molecular and Human Genetics at Baylor College of Medicine. Dr. Lalani cares for TANGO2 warriors in the clinical setting and strongly advocates for patient-centered approaches to care and research. She is also a member of the T2RF Board of Directors and Research Committee, co-leads the TDD Natural History Study and actively supports foundation’s large scale community efforts like the Shine a Light on TANGO2 fundraiser as well as the TANGO2 Family Conference.
Samuel Mackenzie, MD, PhD
Dr. Mackenzie is an Assistant Professor of Child Neurology, Neuromuscular Disease, and Neuroscience at the University of Rochester Medical Center, where he cares for TANGO2 warriors in his clinic. Involved with T2RF since 2020, he has served on the Research Committee, Board of Directors, and as Chair of the Scientific Advisory Board. As a clinician-researcher, Dr. Mackenzie has led and supported multiple foundation-funded studies advancing understanding of TDD, helping to guide future therapies, while mentoring the next generation of researchers committed to the fight against TDD.
Christina Miyake, MD, MS, MPH
Dr. Miyake, a world-renowned TDD clinician-researcher, is a pediatric cardiologist and electrophysiologist, and serves as Director of the Cardiovascular Genetics Arrhythmia Program at Baylor College of Medicine. With support from the foundation, she recently launched a global, 24/7 TDD clinical consult hotline. As a founding member of the T2RF Scientific Advisory Board and lead researcher for the foundation funded TDD Natural History Study, Dr. Miyake combines clinical care, research, and family perspectives to improve outcomes and advance the search for a cure.
Michael Sacher, PhD
Dr. Sacher received his PhD in Biochemistry from McGill University and completed postdoctoral work in the Department of Cell Biology at Yale University. He began his independent research lab at Concordia University in 2006. His work led to the discovery of TRAPP complexes critical for protein transport, and more recently his lab has established model systems to study TANGO2, validating that vitamin B5 alleviates certain cellular defects. Dr. Sacher received funding during the Foundation’s inaugural grant cycle in 2020, joined the T2RF Board of Directors in 2021, and is currently a member of the Scientific Advisory Board.
Innovation in Action
Through the TANGO2 Innovation Fund we are investing in discoveries that brings us closer to understanding and treating TDD, offering hope to families worldwide. Current projects focus on our community’s expressed prioirities:
Understanding Cellular Response to Therapeutics
Every breakthrough starts with someone who believes in the power of research. Your contribution supports studies that explore how TANGO2 cells react to new therapies—critical work that brings us closer to safe, effective treatments and a brighter future for every TANGO2 family.
Optimizing
Nutrition
For many living with TDD, nutrition isn’t just about food, it’s about survival. By supporting this vital area of research, you help scientists develop nutrition-based strategies to prevent crises, control seizures, and reduce the burden of daily symptoms, giving families more stability and hope.
Symptom
Management
TDD can cause severe, unpredictable health challenges like muscle breakdown, irregular heartbeats, metabolic crises and movement disorders. Your support fuels critical research to reduce symptoms, improve daily life, and empower patients and families with the tools they need to thrive.
Quality of Life
We’re developing global health metrics, validating patient- & clinician-reported outcomes, and refining surveys to understand how TDD affects daily life. Drive innovations that improve care, support decision-making & strengthen quality of life for TDD warriors.
Cellular and Molecular Mechanisms Disrupted in TDD
We’re uncovering how the TANGO2 gene drives disease at the cellular level. Your donation fuels this critical research, helping scientists identify targets for treatments and accelerate the path to life-changing therapies.
Development & Characterization of Novel Animal, Cellular, and Computational Models
Novel models give us a window into how TANGO2 affects the body and guide the development of new treatments. Your donation accelerates these discoveries, bringing us closer to breakthroughs that improve lives.
Your support fuels this progress—turning today’s research into tomorrow’s hope for better care, new treatments, and ultimately, a cure.
Rachel Wiratunga Mike Owen Helen Gadsby Shannon Healey Occold Jubillee Baptist Church Old Leamingtonians Rugby Club Dave Wright -MindLabPro Carol Peters in memory of Brian Peters. OL’s Mavericks Touch Rugby Olly Pound Julian and Guy Walker Quentin Oury Andrew Bush Helen Edwards Tim Driffill TANGO2 Titans Alice Bird Jenny Barnes Mega Box Fitness Cristina Ramos Megan LePage and Ben Sellwood Sarah Schoen Chris Lockyer Nonie Toogood Chris and Sue Driffill Derek and Mary Packman Tara Woodward
Rachel Wiratunga Mike Owen Helen Gadsby Shannon Healey Occold Jubillee Baptist Church Old Leamingtonians Rugby Club Dave Wright -MindLabPro Carol Peters in memory of Brian Peters. OL’s Mavericks Touch Rugby Olly Pound Julian and Guy Walker Quentin Oury Andrew Bush Helen Edwards Tim Driffill TANGO2 Titans Alice Bird Jenny Barnes Mega Box Fitness Cristina Ramos Megan LePage and Ben Sellwood Sarah Schoen Chris Lockyer Nonie Toogood Chris and Sue Driffill Derek and Mary Packman Tara Woodward
