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

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Niemann-Pick Type C Disease - Pipeline Insight, 2021

Efficacy and safety of arimoclomol in Niemann‐Pick disease type C: Results from a double‐blind, randomised, placebo‐controlled, multinational phase 2/3 trial of a novel treatment - Mengel - 2021 - Journal of Inherited

NIH clinical trial begins for treatment of rare, fatal neurological disorder

NIH teams with industry to develop treatments for Niemann-Pick Type C disease.

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

Miglustat for treatment of Niemann-Pick C disease: a randomised controlled study - The Lancet Neurology

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

Valley family fights for drug to save sick child

The Neuropsychiatry of Niemann-Pick Type C Disease in Adulthood

Rare Disease Video - Niemann-Pick Disease Types A & B - National Organization for Rare Disorders

Combination therapy a potential strategy for treating Niemann-Pick disease

Rare Disease/Orphan Disease

Adult Onset Niemann-Pick type C (NPC) – NNPDF