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Table 1 Impact scores (n) for selected signs and symptoms by age range and according to families and clinicians’ perspectives

From: Patient reported outcomes for phosphomannomutase 2 congenital disorder of glycosylation (PMM2-CDG): listening to what matters for the patients and health professionals

  1. 1– < 2—No or slight negative impact; 2– < 3—Moderate negative impact; 3– < 4—Negative impact; 4– < 5—Extremely negative impact. q—adjusted p-value
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Orphanet Journal of Rare Diseases

ISSN: 1750-1172