Exploring patient experience with CMS outcome measures
Research type
Research Study
Full title
Exploring the lived experience of patients with Congenital Myasthenic Syndromes (CMS) about assessment tools used clinically and patient views on what might capture meaningful change.
IRAS ID
365346
Contact name
Hayley Ramjattan
Contact email
Sponsor organisation
Oxford University Hospitals NHS Foundation Trust
Duration of Study in the UK
1 years, 0 months, 0 days
Research summary
Congenital Myasthenic Syndromes (CMS) are rare inherited conditions that affect how nerves and muscles work together. People with CMS have muscle weakness which gets worse with activity. Symptoms can be mild or severe, and they often change over time, making the condition hard to assess.
Clinicians and researchers often use tools (known as outcome measures) like timed tests or questionnaires to track changes in a condition or see how well a treatment is working, but there aren’t any tools designed specifically for CMS. Instead, they use tools made for other nerve and muscle conditions, which may not always work well for CMS patients. Unlike Myasthenia Gravis, where patients’ experiences have been studied, little is known about what life is like for people with CMS or how best to measure their symptoms.
This study aims to learn from people living with CMS about their experiences with current assessment tools and what they think would show meaningful change in their condition. This will be done through semi-structured patient interviews and then analyse the responses to find common themes.
The results will help shape a larger project and the development of a more reliable way of measuring the condition - one that reflects what matters most to patients, which can be adopted in clinical and research settings.
REC name
West Midlands - South Birmingham Research Ethics Committee
REC reference
26/WM/0091
Date of REC Opinion
29 Apr 2026
REC opinion
Unfavourable Opinion