At a glance: the largest Niemann-Pick type C1 cohort with 602

Clinical disease progression and biomarkers in Niemann–Pick disease type C: a prospective cohort study, Orphanet Journal of Rare Diseases

Niemann–Pick disease type C Therapeutics Market - NPC Therapeutics Market

Experimental treatment for Niemann-Pick disease type C1 appears safe, effective

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

PDF) At a glance: the largest Niemann-Pick type C1 cohort with 602 patients diagnosed over 15 years

Niemann-Pick Disease Type C: Mutation Spectrum and Novel Sequence Variations in the Human NPC1 Gene

Methods and compositions for treating a proprotein convertase subtilisin kexin (PCSK9) gene-associated disorder Patent Grant Fitzgerald December 1, 2 [Alnylam Pharmaceuticals, Inc.]

Consensus clinical management guidelines for Niemann-Pick disease type C. - Abstract - Europe PMC

PDF) Recommendations for the detection and diagnosis of Niemann-Pick disease type C: An update

The Minderoo-Monaco Commission on Plastics and Human Health - Annals of Global Health

An integrated multiomic approach as an excellent tool for the diagnosis of metabolic diseases: our first 3720 patients

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Diabetic Cardiomyopathy Revisited

Estimated prevalence of Niemann–Pick type C disease in Quebec