SMA Priority Setting

"Putting people
living with SMA
at the heart of
our research programme"

Overview

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:

  1. Question gathering: People were asked to submit their key research questions by way of a survey.
  2. Sorting: The questions were grouped into topic areas and questions that have already been answered were removed.
  3. Prioritising: The remaining questions were sent to people living with SMA, to identify those most important to them.
  4. 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.

The Aim

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’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.

Priority Setting TeamMeet Véronique and complete our survey!

Results

List of the Top 10 unanswered research questions as voted per PSP process

:
1. Can nerve cells (especially motor neurons) be regenerated in people with SMA?
:
2. Are there biological markers (biological molecules in blood or other tissues) that can indicate the progression of SMA?
:
3. Can damaged muscles be rebuilt in people with SMA? How can muscles be strengthened?
:
4. Do people with SMA benefit from a specialised diet (e.g. amino acid diet) or dietary changes such as treatment with supplements?
:
5. What is the best physiotherapy approach for people with SMA? Does it need to be tailored to the individual (e.g. according to age and type of SMA)? Does it need to change during treatment with one of the new drugs?
:
6. What parts of the body are affected by SMA other than nerves and muscles?
:
7. Does electrical stimulation of nerves and/or muscles benefit people with SMA?
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8. What is the best approach to treating and preventing problems with bones and joints (orthopaedic problems) for people with SMA?
:
9. What are the best ways to treat and manage fatigue in people with SMA?
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10. How can new technology and assistive devices help people with SMA and their carers?

In a nutshell

Areas with high needs

:
1. Regeneration of NM system
:
2. Biomarkers
:
3. Metabolism / Diet
:
4. Physiotherapy
:
5. Pathways, cells and tissues affected by SMA
:
6. Bones/Joints
:
7. Fatigue
:
8. Innovative technology