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Fig. 3 | Orphanet Journal of Rare Diseases

Fig. 3

From: Stable or improved neurological manifestations during miglustat therapy in patients from the international disease registry for Niemann-Pick disease type C: an observational cohort study

Fig. 3

Annual progression rate in patients who received continuous miglustat therapy between enrolment and last follow up. Data are means ± 95 % CI; no 95 % CI could be calculated for the early infantile-onset group due to the low number of patients. EI, early-infantile onset; LI, late-infantile onset; JUV; juvenile onset; AA, adolescent/adult onset. Note: six patients had insufficient disability scale data.

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Orphanet Journal of Rare Diseases

ISSN: 1750-1172