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Last Updated: 01/06/2023

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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 parts of the body. There is no cure for NPC, and symptoms only get worse over time. Symptoms can include seizures, difficulty moving or talking, or dementia. But symptoms can vary among different people with the disease. Some may have seizures, while ot...

A Pilot, Randomized, Placebo-Controlled Trial Evaluating the Treatment of Premenstrual Dysphoric Disorder With Oral Contraceptives in Bipolar Disorder.

Summary: This study is a pilot, randomized, placebo-controlled trial evaluating the treatment of Premenstrual Dysphoric Disorder comorbid with Bipolar Disorder using combined oral contraceptives. Lay Summary: This study is being done with the hope of finding a safe and effective treatment for individuals who experience both bipolar disorder and severe premenstrual symptoms. As part of this clinical trial, ...

Evaluation of Biochemical Markers and Clinical Investigation of Niemann-Pick Disease, Type C

Summary: 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 Disease, Type C (NPC), a genetic disorder that results in progressive loss of nervous system function. The study will: 1) look for a clinical or biochemical marker that can be used as a measure of response to treatment, and 2) define the rat...

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Last Updated: 01/06/2023