The results of a European survey on the expectations of clinical trials of 1147 people with facioscapulohumeral myopathy living in 26 European countries including France showed that :
- respondents to the survey are mainly people with FSHD1 (68%) against 7% with FSHD2, the others not having a genetic diagnosis; they are on average 50 years old
- A large half use walking aids or a wheelchair.
- The first symptoms began for 40% of them between the ages of 11 and 20, with a detachment of the shoulder blades (31%), then weakness of the lower limbs (22%) and upper limbs (19%).
- The average time to diagnosis after the first symptoms appeared was 7.9 years.
- Physiotherapy, occupational therapy, exercises and mobility aids are the means most used by patients to improve their health.
- The main motivating factors for participating in a clinical trial are to be able to benefit from the treatment, to have access to the results of the trial and to provide benefits to FSHD patients collectively,
- On the other hand, the distance and accessibility of the investigating centre of the trial as well as the fear of having side effects are barriers to participation in a clinical trial.
- Patients also stressed the importance of having clear information throughout their participation in the clinical trial and feedback on the results of the trial.
These data may help in setting up future clinical trials to encourage the involvement of FSHD patients.
