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

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Leading Life Science Syndicate Commits $25 Million to Series A Funding to Launch Vtesse, Inc., the First Rare Disease Company Spun Out of Cydan Development, Inc.

Niemann-Pick Type C Disease - Pipeline Insight, 2021

Frontiers Study design challenges and strategies in clinical trials for rare diseases: Lessons learned from pantothenate kinase-associated neurodegeneration

IJMS, Free Full-Text

Prevalence of dysphagia in Niemann-Pick disease type C (NP-C) and other

Potential treatment for Niemann-Pick type C, a rare neurodegenerative disease

Orphazyme's Drug for Rare Metabolic Disease Aces Clinical Trial

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

Clinical patient groups with an increased risk of Niemann-Pick disease

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

Drug Repurposing for Rare Diseases: Trends in Pharmacological Sciences

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