Skip to main content
Fig. 2 | Orphanet Journal of Rare Diseases

Fig. 2

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. 2

Proportions of patients who received continuous miglustat therapy during follow up* and were categorized as ‘improved/stable’. *The length of the observation period varied for each patient; data values based on all patients with available data, per age at neurological onset subgroup. EI, early-infantile onset; LI, late-infantile onset; JUV; juvenile onset; AA, adolescent/adult onset. Note: six patients had insufficient disability scale data

Back to article page

Orphanet Journal of Rare Diseases

ISSN: 1750-1172