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

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Orphan & Rare Disease

Niemann-Pick Disease Drug Type C Treatment Market Size

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

Niemann-Pick Disease Type C

The Niemann-Pick Foundation finances a clinical trial of a drug to stop the cognitive deterioration of the disease – IDIBELL

TRIALS: A Fight for Children with Niemann-Pick Type C, a Fatal Genetic Disease

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

Frontiers Case Report: Be Aware of “New” Features of Niemann–Pick Disease: Insights From Two Pediatric Cases

Niemann-Pick disease type C Alzheimer Society of Canada

5 Most Promising Companies offering hope to patients with Niemann-Pick Type C

Case Studies, Clinical Development Case Studies