Table 1. Route of administration and dose of HPβCD administered to 11 groups of cats.
GenotypeRoute of
DoseDosing intervalAnimal numbersSurvival time mean and
SD, or longest (weeks)
No mutant allele
(normal control)
NPC1UntreatedUntreatedUntreated221720.7 ± 5.0
NPC1SC1000 mg/kg and
25 mg/kg
7 days5121.8 ± 6.5
NPC1SC4000 mg/kg7 days0231, 36
NPC1SC8000 mg/kg7 days3235.2 ± 12.4
NPC1IC3.8 mg14 days1249
NPC1IC7.5 mg14 days1262
NPC1IC15 mg14 days2166
NPC1IC30 mg14 days15>76*
NPC1IC60 mg14 days21>76*
NPC1IC120 mg14 days27>76*
NPC1IC, SC120 mg, 1000 mg/kg14 days, 7 days53>76*
NPC1IC120 mg postsymptomatic14 days4443.5 ± 5.8

*No cats in this group died of NPC disease during the study period of 76 weeks. †Longest survival time of individual cats that died due to signs of NPC disease. Remaining cats in this group were euthanized at ~24 weeks of age to collect histological data. ‡Treatment first began at 16 weeks of age.