Professor Paul Maddison and Dr Phil Ambrose will study deep phenotyping and immune profiling in autoimmune myasthenia.

To date, almost all studies of myasthenia have collected data on clinical features and antibody results from patients from multiple centres or at different times of their illness, which can introduce bias.

Over the last 3 years, initial funding from myaware has allowed Professor Paul Maddison and his team to recruit over 150 patients with myasthenia from Nottingham, Birmingham and Oxford from diagnosis. This unique collection of patients has allowed them to track every symptom in every patient over time, such that they are building up an entire picture of symptoms in each patient with myasthenia.

The data they are collecting will allow them to predict more accurately the nature of myasthenia over time in individual patients and as a group, and also to compare late-onset (over the age of 50 years) with early-onset myasthenia with unbiased accuracy. They are also collecting quality of life measures each year to see how the illness impacts on each patient and their home life.

They will also be able to see whether national guidelines published in 2015 for the treatment of myasthenia are making a difference compared to previously audited UK data on hospital admissions for myasthenia.

This new project funded by myaware will allow the team to complete follow-up in all their patients for 2-3 years to obtain definitive unbiased clinical outcomes in all patients. In addition, they will be able to see how antibody levels from diagnosis, and yearly thereafter, reflect a patient’s condition.

Finally, they hope to match these clinical characteristics with the function of the immune system in both early and late onset myasthenia. They will be working with Oxford and Yale Universities to look at the defective immune cells from the study patients to see where in the pathway of the immune system the problem starts that causes the antibodies. This includes studying not only the cells producing the main and commonest antibody to the acetylcholine receptor, but also antibodies to MuSK and antibodies to voltage gated calcium channels, as found in Lambert-Eaton myasthenic syndrome (LEMS).


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