ENDECE for Niemann-Pick Disease Type C (NPC1)
NDC-1308 (MC2) has a Dual Mechanism of Action (MOA) that may both slow the progression of Niemann-Pick disease type C (NPC1) and potentially reverse the functional losses associated with NPC1. Using a nebulizer, NDC-1308 (MC2) will be directly delivered to the central nervous system to reduce inflammation, and remyelinate patients with the two key pathologies that drive NPC1.
- Chronic Progressive Neuroinflammation
- Demyelination / Hypomyelination
NPC1: A RARE PROGRESSIVE DISEASE
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.
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
HOW NDC-1308 (MC2) WORKS FOR NPC1
Directly Polarizes Macrophages to the M2 Anti-inflammatory State
Directly Induces Remyelination