Research strategy
The Tuberous Sclerosis Association’s research strategy for funding Tuberous Sclerosis Complex research
The Tuberous Sclerosis Association (TSA) is proud of our position as a world-leader in Tuberous Sclerosis Complex (TSC) research.
From improving diagnosis of TSC to trialling new strategies and medicines for managing the condition, funding new research has always been a key priority for us.
This strategy marks an important milestone. It has been shaped through extensive consultation with researchers, clinicians, and, most importantly, the wider TSC community. We are deeply grateful to everyone who has contributed their time, insight, and experience to help ensure the strategy reflects both our ambition and the practical realities of advancing research in TSC.
The TSA’s priorities for research into TSC are:
Animation courtesy of Paul Davis
To refresh our research strategy, we undertook a three-phase process designed to ensure broad input from the TSC community and expert oversight
To achieve our research vision, we know it is not enough to set priorities, we must be clear about how we will deliver them.
These enablers are the essential things we will focus on to make our ambitions a reality. They provide the structure, support, and momentum that will allow us to accelerate progress, strengthen research, and ensure that everything we do is grounded in collaboration and the lived experience of people with TSC.
Collaborating for impact
Partnership is at the heart of progress. We will work with researchers, clinicians, funders, industry, and international networks to combine expertise and resources, ensuring that research into TSC achieves more than any organisation could deliver alone.
Embedding lived experience
The voices of people affected by TSC are essential in shaping research. From setting priorities to designing studies and participating in trials, we will ensure that lived experience guides and strengthens research at every step through the TSC Research Volunteer Network.
Driving innovation
We will embrace new ideas, technologies, and approaches that have the potential to transform TSC research. By supporting bold and creative science, and by building capacity in the research community, we will accelerate progress towards our research vision.
The TSA is committed to funding research in line with its three research priorities. Our vision is a world where TSC and its effects are conquered.
Our research priorities from 2025-2028 are:
1. To deepen understanding of the lifelong impact of TSC on individuals and families, including:
1.1. Understanding adulthood in TSC: through research we will understand how TSC affects individuals across the adult lifespan, and identifying the biological, psychological, and social factors that influence health, wellbeing, and long-term outcomes
1.2. Investigating hormonal stages: through research we will explore how key hormonal stages — such as puberty, pregnancy, and menopause — influence progression, symptoms, and wellbeing in people with TSC
1.3. Identify early life predictors: through research we will use tools such as natural history databases to uncover behaviours, symptoms, and biomarkers in early life that shape long-term outcomes and quality of life for people with TSC
2. To ensure that people with TSC benefit from the best possible treatments, including:
2.1. Improving outcomes for TSC-Associated Neuropsychiatric Disorders (TAND): through research we will explore pharmacological, behavioural, educational, and psychosocial interventions to improve outcomes for people living with the TAND
2.2. Optimising epilepsy therapies: through research we will make the best use of existing and emerging therapies to improve epilepsy management in TSC, reducing seizures and improving quality of life
2.3. Better understanding treatments in practice: through research we will build knowledge about the effectiveness, tolerability, and interactions of treatments already used in TSC, ensuring real-world evidence guides better care and outcomes
2.4. Exploring gene-based therapies: through research we will investigate the potential of gene-based approaches to treat or modify TSC, laying the groundwork for future breakthroughs and transformative treatments
3. To ensure that everyone with TSC has access to fair, consistent, and effective care throughout their lives, including:
3.1. Understanding barriers to care: through research we will identify the challenges that prevent fair and consistent service delivery for people with TSC, and developing evidence-based solutions to improve access and equity
3.2. Optimising surveillance: through research we will define the most effective ways to monitor TSC across the lifespan, including the right approaches, tools, and intervals to deliver the best outcomes
3.3. Harnessing digital technologies: through research we will use digital tools and innovations to transform how TSC is monitored and managed, while supporting independence and improving quality of life
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