NIEMANN-PICK DISEASE TYPE C (NPC1)
Niemann-Pick disease type C (NPC1) is a rare genetic disorder in which gene mutations are passed from parents to children in a pattern called autosomal recessive inheritance. Patients with NPC1 are unable to transport cholesterol and fatty substances (lipids) out of lysosomes causing an accumulation of these compounds leading to progressive and lethal neurodegeneration.
NPC1: A RARE PROGRESSIVE DISEASE
Niemann-Pick disease type C (NPC1) primarily affects children, but can occur at any age. Symptoms Include:
Clumsiness and difficulty walking
Excessive muscle contractions (dystonia) or eye movements
Difficulty swallowing and eating
ENDECE’S APPROACH TO NPC1
Due to its Dual Mechanism of Action (MOA), NDC-1308 may both slow the progression of Niemann-Pick disease type C and potentially reverse the functional losses associated with NPC1 in children.