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

Niemann–Pick Disease Type C: Induced Pluripotent Stem Cell–Derived Neuronal Cells for Modeling Neural Disease and Evaluating Drug Efficacy - Daozhan Yu, Manju Swaroop, Mengqiao Wang, Ulrich Baxa, Rongze Yang, Yiping Yan, Turhan

Recent advances in the treatment of Niemann pick disease type C: A mini-review - ScienceDirect

Recommendations for the detection and diagnosis of Niemann-Pick disease type C

Miglustat in Niemann-Pick disease type C patients: a review, Orphanet Journal of Rare Diseases

Effects of miglustat therapy on neurological disorder and survival in early-infantile Niemann-Pick disease type C: a national French retrospective study, Orphanet Journal of Rare Diseases

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

Niemann-Pick disease type C

Niemann–Pick disease - Wikipedia

Unbiased yeast screens identify cellular pathways affected in Niemann–Pick disease type C

5 Most Promising Companies offering hope to patients with Niemann-Pick Type C

Miglustat in patients with Niemann-Pick disease Type C (NP-C): A