Dutch researchers have designed a follow-up protocol for patients suffering from facioscapulohumeral muscular dystrophy (FSHD) in order to gain a better understanding of the natural history of this disease:
- 154 symptomatic patients confirmed by molecular biology were included in the study,
- the median age of the cohort was 51 years,
- a battery of tests and functional scores (MFM, 6-minute walk test, manual testing, etc.) was used,
- a small but statistically significant reduction was observed after five years of observation.
However, since the progression of the disease was minimal over five years, the tools used in the study do not appear to be suitable for clinical trials, which usually last two years, suggesting that the duration of the trials should be extended or the results re-evaluated.