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

Sleep disturbances

Difficulty swallowing and eating

Recurrent pneumonia

ENDECE’S APPROACH TO NPC1

NDC-1308

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.