Experimental treatment for Niemann-Pick disease appears safe

An experimental drug has appeared to slow the progression of Niemann-Pick disease the drug 2-hydroxypropyl-beta-cyclodextrin is being tested

A Practical Chromogenic Procedure for the Detection of Homozygotes and Heterozygous Carriers of Niemann-Pick Disease

Potential pharmacological strategies targeting the Niemann-Pick C1 receptor and Ebola virus glycoprotein interaction - ScienceDirect

Lithium ameliorates Niemann-Pick C1 disease phenotypes by impeding STING/SREBP2 activation - ScienceDirect

IJMS, Free Full-Text

Anti-Niemann Pick C1 Single-Stranded Oligonucleotides with Locked Nucleic Acids Potently Reduce Ebola Virus Infection In Vitro: Molecular Therapy - Nucleic Acids

Valley boy battling rare disease getting stronger

Niemann-Pick Disease Concise Medical Knowledge

Parents of twins with Niemann-Pick find a treatment

Dermatologic Manifestations of Niemann-Pick Disease: Background, Pathophysiology, Etiology

Zevra Therapeutics – NNPDF

Families plead with FDA for drug to treat Niemann-Pick Disease Type C

Emerging Startups Take Aim at Lysosomal Storage Disorders

Inhibition of Histone Deacetylases 1, 2, and 3 Enhances Clearance of Cholesterol Accumulation in Niemann-Pick C1 Fibroblasts