Rare Diseases Research: Clinical Trial for Niemann-Pick Type C

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Newborn screening test developed for rare, deadly neurological disorder – Washington University School of Medicine in St. Louis

Laboratory diagnosis of Niemann-Pick disease type C: the filipin staining test.

Researchers find immune component to rare neurodegenerative disease : Newsroom - UT Southwestern, Dallas, Texas

Diagnostic workup and management of patients with suspected Niemann-Pick type C disease - Apostolos Papandreou, Paul Gissen, 2016

New formulation of FDA-approved drug may help treat Niemann-Pick Type C disease, News, Notre Dame News

Niemann Pick Disease - an overview

Think Again. Think NP-C

Understanding the phenotypic variability in Niemann-Pick disease type C (NPC): a need for precision medicine

Australian Research Updates Australian NPC Disease Foundation Inc

Rare Diseases Research: Clinical Trial for Niemann-Pick Type C

IJMS, Free Full-Text

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

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

Niemann-Pick disease type C clinical database: cognitive and coordination deficits are early disease indicators – topic of research paper in Clinical medicine. Download scholarly article PDF and read for free on CyberLeninka

University College London begins preclinical studies to develop a treatment for Niemann Pick C disease with support from the Columbus Foundation – Columbus Children's Foundation