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.

Speaker Series - Niemann-Pick Disease Type C

Niemann-Pick type C disease – the tip of the iceberg? A review of

Niemann-Pick disease type C Orphanet Journal of Rare Diseases

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

Potential treatment for Niemann-Pick type C, a rare

Miglustat for treatment of Niemann-Pick C disease: a randomised

Recommendations for the detection and diagnosis of Niemann-Pick

Bloomsbury Genetic Therapies Outlines Pre-Clinical Data on CNS

Niemann Pick Disease Type C - Symptoms, Causes, Treatment

Identification of Novel Biomarkers for Niemann–Pick Disease Using