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Table 4 Efficacy analysis based on hypothetical treated population of at least 70 patients

From: The SPARKLE registry: protocol for an international prospective cohort study in patients with alpha-mannosidosis

Expected proportion

95% CI lower limit

95% CI upper limit

95% actual width

0.60

0.476

0.715

0.239

0.65

0.527

0.760

0.234

0.70

0.579

0.804

0.225

0.75

0.632

0.846

0.214

0.80

0.687

0.886

0.199

  1. CI confidence interval
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Orphanet Journal of Rare Diseases

ISSN: 1750-1172