Reporting preclinical gene therapy studies in the field of Niemann-Pick type C disease according to the ARRIVE guidelines

Table 4 Humane endpoints

Parameter	Observation	References
Weight loss	15% loss of body weight after a 24 h period	[16]
	30% weight loss of maximum weight	[11]
	> 15% loss of body weight in a week	[14]
	Loss of 1 g of body weight within a 24 h period	[15]
	Rapid weight loss	[10]
Locomotor function	Reluctance to move, repeated falling to the side during forward ambulation	[11]
	Tremor, abnormal gait, unbalanced	[14, 22]
	Severe loss of motor function	[10]
Skin and fur	Ruffled fur, yellow coat	[22]
Eyes	Palpebral closure/eyes appearing dull rather than bright	[11]
General appearance	Penile prolapse	[14]

ISSN: 1750-1172