Monitoring, either as part of the usual follow-up of patients with muscular dystrophy or as part of clinical trials, is largely based on the use of functional scores:
- researchers report, in association with Duchenne muscular dystrophy (DMD) patient associations, the development of an upper limb function scale centred on the needs and feelings of patients themselves,
- the correlation of the results with the existing tool, the PUL 2.0, proved excellent.
- other American researchers have successfully developed a new scale for facioscapulohumeral muscular dystrophy (FSHD) entitled FSH-HI (for FSH-Health-Index), also based on input from patients themselves.
It remains to be seen whether these new tools can be used to design future therapeutic trials in both DMD and FSHD.
Patient reported outcome measure for upper limb in Duchenne muscular dystrophy: correlation with PUL2.0. Cicala G, Pane M, Coratti G et al. Neuromuscul Disord. 2023 Sept.
The Facioscapulohumeral Muscular Dystrophy-Health Index: Development and evaluation of a disease-specific outcome measure. Varma A, Weinstein J, Seabury J et al. Muscle Nerve. 2023 Oct.
