Setting research priorities
SMA Europe partnered with the James Lind Alliance to identify the top 10 unanswered research questions on SMA from people living with the condition, their caregivers and healthcare providers.
This initiative involved a number of steps:
- Question gathering: People were asked to submit their key research questions by way of a survey.
- Sorting: The questions were grouped into topic areas and questions that have already been answered were removed.
- Prioritising: The remaining questions were sent to people living with SMA, to identify those most important to them.
- Narrowing down to 10: A group of people with SMA, family members and health care professionals took part in a workshop and were asked to narrow the most voted-for questions to our top 10.
Why this Priority Setting initiative?
By hearing the research priorities of our community, we can inform our research programme, push other funders, scientists and service providers to recognise the views and needs of those living with SMA and get their questions answered.
We are committed to funding high quality research that answers the key questions we have as a community. We are doing this in a number of ways already, including funding new research projects every other year through our Call for Research Proposals.
The scope of the SMA PSP was on living with SMA, its management and care, as well as treatments. Excluded from the scope were diagnosis and newborn screening.
Who was involved?
The project was led by our Research Programme Manager, with the help of a James Lind Alliance advisor and an Information Specialist.
The project’s steering group was composed of people who live with SMA, their carers, an ENMC representative, SMA Europe advisors and relevant health and social care professionals - a neurologist, two physiotherapists, a clinical dietician, a speech and language therapist and a clinical neuro-psychologist.
- The James Lind Alliance
- Our Steering Committee
This project is funded by SMA Europe.