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Effectiveness of asfotase alfa for treatment of adults with hypophosphatasia: results from a global registry.

Orphanet J Rare Dis · 2024 · PMC10921796 · PMID 38459585

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Similarly, mean changes in physical functioning score (change: 4.10 [95% CI: 0.20, 8.00; P = 0.04]), physical role functioning score (change: 3.69 [95% CI: 0.19, 7.18; P = 0.039]), and bodily pain score (change: 4.52 [95% CI: 0.34, 8.70; P = 0.035]; n = 28) were statistically significant after 12 months of treatment (n = 28), but changes did not reach statistical significance through 36 months.

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