Evolution of mobility, pain/discomfort, self-care, and mental health in patients with alpha-mannosidosis: an international caregiver and patient survey.

Orphanet J Rare Dis · 2025 · PMC12057280 · PMID 40336024

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a slight trendno p-value reported

Overall changes HSCT patients reported the lowest mean VAS score 5 years ago (1.6 ± 1.5; Supplementary Table 1 ) compared to patients on ERT and UP, a trend that continued for now, however, only patients receiving ERT reported a slight trend towards improvement in pain (-0.2 ± 2.0) (Fig. 3 a).

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