Professional
I’m a professional
For research teams, clinicians, patient organizations, funders and development teams: explore the research approach, possible project outputs and ways to contribute your science.
Explore the research approachTwo perspectives. One purpose.
Where would you like to begin?
Patient or caregiver
I’m a patient or caregiver
For people living with rare conditions, parents, families and caregivers: see how lived experience can shape research, explore science in plain language and find community resources.
Explore lived experienceOur direction
Better questions begin with the whole person.
A condition has a biological story and a lived story. Our developing approach brings them together: understand what is known, learn what matters, choose meaningful measures, examine patterns and build a practical research plan.
Evidence in Context · In development
Our research initiative is initially focused on neurodevelopmental conditions. We are developing the questions, methods and collaborations that could support this work.
Science Notes
A clearer way into the evidence.
Measurement
What makes a measure trustworthy?
A precise number still needs to answer a meaningful question.
Read the noteUnderstanding evidence
A pattern is a starting point
Why a relationship between two things does not, on its own, establish cause.
Read the noteDaily life
The story between appointments
How lived experience can help shape natural history research.
Read the note