Adult Onset Niemann-Pick type C (NPC) – NNPDF
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Investigational New Drug (IND) - The Addi and Cassi Fund
Understanding the phenotypic variability in Niemann-Pick disease type C (NPC): a need for precision medicine
At a glance: the largest Niemann-Pick type C1 cohort with 602 patients diagnosed over 15 years
Estimating the prevalence of Niemann-Pick disease type C (NPC) in the United States - ScienceDirect
Genotype-phenotype relationship of Niemann-Pick disease type C: a possible correlation between clinical onset and levels of NPC1 protein in isolated skin fibroblasts
Niemann Pick C Awareness Group
Niemann Pick Disease Type C - Symptoms, Causes, Treatment
Validation of the 5-domain Niemann-Pick type C Clinical Severity Scale
Niemann-Pick disease type C, Orphanet Journal of Rare Diseases
Do heterozygous mutations of Niemann–Pick type C predispose to late-onset neurodegeneration: a review of the literature
Health insurance literacy and health services access barriers in Niemann–Pick disease: the patient and caregiver voice, Orphanet Journal of Rare Diseases
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