Evolution of structural neuroimaging biomarkers in a series of adult patients with Niemann-Pick type C under treatment

Table 2 Clinical evolution of NPC patients using the NPC composite score [12]

Patient	1	2	3	4	5	6	7	8	9	10	11
Baseline NPC score	10	10	9	7	3	5	10	11	10	10	15
Last NPC score	10	13	8	10	8	5	10	12	10	10	15
Time range (year)	5	9	4	4	4.5	1	9	2	5	2	9
Sustained treatment	+	+	+	-	-	+	+	+	+	+	+

Eleven patients had a clinical follow-up ranging from 1 to 9 years. Clinical stabilization was defined as a variation in the total score ≤ 1. Patients 2, 4 and 5 worsened, but only patient 2 had not interrupted treatment with miglustat

ISSN: 1750-1172