Why the study?
Prior studies may have overestimated wheelchair use in FSHD1 by relying on patient reports rather than objective assessments, whereas future trials need accurate disability data.
In a large Chinese cohort of genetically confirmed FSHD1 patients, the incidence of wheelchair use was 8.9%, with smaller D4Z4 repeat lengths and earlier onset age predicting faster progression to wheelchair dependence.
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Does not support practice changes in FSHD1; extends observational data on progression predictors but leaves generalizability open.
Qiu et al. (2022) studied this question.
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