Table 1 .

Abstract background, page 1:

cholestane-3β,5α-,6β-triol

cholestane-3β,5α,6β-triol

Abstract results, page 1, lines 4-7:

Compared with healthy individuals, the NPC population had significantly lower levels of cholesterol esterification (p < 0.0001), HSP70 (p < 0.0001) and skin unesterified cholesterol (p = 0.0006). Cholestane-triol levels were significantly higher in individuals with NPC versus healthy individuals (p = 0.008) and correlated with the 5-domain NPCCSS (Spearman’s correlation coefficient = 0.265, p = 0.0411).

Compared with healthy individuals, the NPC population had significantly lower levels of cholesterol esterification (p < 0.0001) and HSP70 (p < 0.0001), and significantly higher levels of skin unesterified cholesterol (p = 0.0006). Cholestane-triol levels were also significantly higher in individuals with NPC versus healthy individuals (p = 0.008) and were correlated with the 5-domain NPCCSS (Spearman’s correlation coefficient = 0.265, p = 0.0411).

Abstract trial registration, page 2:

2014–005,194-37

2014-005193-37

Background, page 2, paragraph 2, line 9:

that affect gross motor skills, swallowing ability cogni-

that affect gross motor skills, swallowing ability and cogni-

Results, page 4, paragraph 4, line 11:

tion still had an end-of-trial visit and provided data for

tion still had an end of study visit and provided data for

Results, page 4, paragraph 6, line 3:

by referring clinicians as current medical conditions, was

by referring clinicians as current medical conditions, were

Results, page 4, paragraph 7, line 9:

(Fig. 3b).

(Fig. 3c).

Figure 3 legend, line 4,

an end-of-trial visit and is included in efficacy assessments

an end of study visit and is included in efficacy assessments

Table 3, row 1:

Total number of different alleles

Total number of distinct alleles

Table 4, title:

Change in disease biomarkers over the 6–14-month observation period compared with those of healthy individuals

Disease biomarkers over the 6–14-month observation period and those of healthy individuals

Results, page 8, paragraph 4, line 20:

(Table 6).

(Table 7).

Figure 5 legend, line 1-2:

a. PBMC unesterified cholesterol. b. Skin unesterified cholesterol.

a. Skin unesterified cholesterol. b. PBMC unesterified cholesterol.

Table 5, line 20:

Serious adverse events [indented]

This is incorrectly indented, please align to the left.

Table 6

Table 7

Table 7

Table 6

Results, page 11, paragraph 2, line 12:

(Table 7).

(Table 6.)

Table 7, heading:

MEAN 5-domain NPCCSS score (±SD)

Mean 5-domain NPCCSS score (±SD)

Discussion, page 12, paragraph 1, line 12-13:

date and to confirm the patients and clinicians view on

clinical meaningful changes on the 5-domain NPCCSS

date and to confirm the patients’ and clinicians’ views on

clinically meaningful changes on the 5-domain NPCCSS

Discussion, page 13, paragraph 2, line 12:

tane burden with NPC disease severity [22]. However,

tane triol burden with NPC disease severity [22]. However,

Discussion, page 13, paragraph 3, line 19:

this was likely owing to the limited viability of PBMCs

this was likely due to the limited viability of PBMCs

Discussion, page 14, paragraph 3, line 11:

uting to the 5-domain NPCCSS were locally independent

uting to the 5-domain NPCCSS was locally independent

Methods, page 15, paragraph 2, line 9:

Incorrect formatting of citation (15)

Delete

Methods, page 16, paragraph 6, line 22:

Incorrect formatting of abbreviation and citation (ICC2,13)

(ICC) [2,13]

Abbreviations:

MCID: Minimal clinically important difference;

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