Rare Diseases Research: Clinical Trial for Niemann-Pick Type C
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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
Niemann–Pick type C disease: cellular pathology and pharmacotherapy - Wheeler - 2020 - Journal of Neurochemistry - Wiley Online Library
Cyclo Therapeutics announces positive Niemann-Pick disease from small study - Drug Discovery and Development
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Niemann-Pick Type C News Dana's Angels Research Trust (DART)
Frontiers Study design challenges and strategies in clinical trials for rare diseases: Lessons learned from pantothenate kinase-associated neurodegeneration
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Niemann-Pick Disease, Type C1 disease: Malacards - Research Articles, Drugs, Genes, Clinical Trials
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Recent advances in the treatment of Niemann pick disease type C: A mini-review - ScienceDirect
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At a glance: the largest Niemann-Pick type C1 cohort with 602 patients diagnosed over 15 years
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PDF] Diagnostic tests for Niemann-Pick disease type C (NP-C): A critical review.
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