Skip to main content
Fig. 1 | Orphanet Journal of Rare Diseases

Fig. 1

From: Disease characteristics, prognosis and miglustat treatment effects on disease progression in patients with Niemann-Pick disease Type C: an international, multicenter, retrospective chart review

Fig. 1

Qualitative description of first event*, diagnosis, miglustat initiation, last contact, and death in (a) Infantile, (b) Juvenile and (c) Adult age groups. *First event was defined as the first sign or symptom that is included in the 6-domain unified disability scale. From the EDC medical history, first sign or symptom could be either neurologic, visceral, or psychiatric. EDC, electronic data capture

Back to article page

Orphanet Journal of Rare Diseases

ISSN: 1750-1172