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Evaluation of Biochemical Markers and Clinical Investigation of Niemann-Pick Disease, Type C
This study will evaluate clinical and laboratory tests that might be useful in determining if an investigational drug can slow the progression of Niemann-Pick …
Niemann-Pick Disease, Type C
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)NCT00344331
Phase 3
Effects of N-Acetyl-L-Leucine on Niemann-Pick Disease Type C (NPC): A Phase III, Randomized, Placebo-controlled, Double-blind, Crossover Study
A pivotal, randomized, double-blind, placebo controlled, multi-center therapeutic study for patients age 4 and older with a confirmed diagnosis of Niemann Pick…
Niemann-Pick Disease, Type C
IntraBio IncNCT05163288
Establishment of Genomic and Phenotypic Database for Niemann-Pick Disease, Type C
Background: Niemann-Pick type C (NPC) disease is a rare, progressive neurodegenerative disease that affects mainly the brain, liver, and spleen but also other…
Niemann-Pick Disease, Type C
Compensation
Up to $190
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)NCT05588167