Therapeutic intervention for Niemann–Pick type C disease must target

Effect of acid sphingomyelinase deficiency in type A Niemann-Pick disease on the transport of therapeutic nanocarriers across the blood–brain barrier

Adult Niemann-Pick disease type C in France: clinical phenotypes and long-term miglustat treatment effect, Orphanet Journal of Rare Diseases

Tolerance of chronic HDACi treatment for neurological, visceral and lung Niemann-Pick Type C disease in mice

Niemann Pick Disease Type C - Symptoms, Causes, Treatment

New murine Niemann-Pick type C models bearing a pseudoexon-generating mutation recapitulate the main neurobehavioural and molecular features of the disease

Niemann- Pick Disease Type C (NPC) Market Report 2023-2033 - Wissen Research

Identification of Niemann-Pick C1 protein as a potential novel SARS-CoV-2 intracellular target - ScienceDirect

Frontiers Adult-Onset Niemann–Pick Disease Type C: Rapid Treatment Initiation Advised but Early Diagnosis Remains Difficult

Liposome-targeted recombinant human acid sphingomyelinase: Production, formulation, and in vitro evaluation - ScienceDirect

Finding pathogenic commonalities between Niemann-Pick type C and other lysosomal storage disorders: Opportunities for shared therapeutic interventions - ScienceDirect

Niemann-Pick Disease Type C (NP-C) Gene Therapy Activation Meeting (GAM) - International Niemann-Pick Disease Alliance (INPDA)