Potential treatment for Niemann-Pick type C, a rare neurodegenerative disease

Researchers in Japan have improved a potential treatment for a rare genetic disease, decreasing its negative toxic effects by threading it onto a dumb-bell-shaped chain and holding it in place until it reaches its target.

Adult Niemann-Pick disease type C in France: clinical phenotypes

Finding pathogenic commonalities between Niemann-Pick type C and

KemPharm - KemPharm Reports Third Quarter Financial Results and

IJMS, Free Full-Text

Identification of Novel Biomarkers for Niemann–Pick Disease Using

Estimating the prevalence of Niemann-Pick disease type C (NPC) in

IJMS, Free Full-Text

Do heterozygous mutations of Niemann–Pick type C predispose to

Myelin Defects in Niemann–Pick Type C Disease: Mechanisms and

Niemann-Pick type C: contemporary diagnosis and treatment of a

Therapeutic intervention for Niemann–Pick type C disease must

Rare Disease Video - Niemann-Pick Disease Types A & B - National

IJMS, Free Full-Text