Table 1.

Baseline Characteristics of the Patients and the Drug Product.

Characteristic	Value
Patients
No. enrolled*	17
Age at enrollment (yr)
Median	6
Range	4–13
Loes score†
Median	2.0
Range	1.0–7.5
Score on neurologic function scale‡
Median	0
Range	0
Time from consent to infusion of drug product (days)
Median	67.0
Range	58.0–89.0
Drug product
Vector copy number (vector copies/diploid genome)
Median	1.0
Range	0.5–2.5
Dose (CD34+ cells/kilogram of body weight)
Median	10,500,000
Range	6,000,000–19,400,000

^*All the patients were male.

^†Loes scores range from 0 to 34, with higher scores indicating an increased extent of lesions on magnetic resonance imaging.

^‡Scores on the cerebral adrenoleukodystrophy–specific neurologic function scale range from 0 to 25, with higher scores indicating more severe deficits. The scale is used to evaluate the severity of gross neurologic dysfunction through an assessment for 15 disabilities across multiple domains; a score of 0 indicates the absence of clinical signs of cerebral disease.