Niemann-Pick disease

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Niemann-Pick disease

[′nē‚män ′pik di‚zēz]
(medicine)
A hereditary sphingolipidosis due to an enzyme deficiency resulting in abnormal accumulation of sphingomyelin; symptoms include anemia, enlargement of the liver, spleen, and lymph nodes, gastrointestinal disturbances, and various neurologic deficits. Also known as lipid hystiocytosis.
References in periodicals archive ?
Biotechnology company CTD Holdings Inc (Other OTC:CTDH) reported on Thursday that it has started enrolling patients under its Phase I clinical study in the US to evaluate the intravenous administration of Trappsol Cyclo in patients with Niemann-Pick Disease Type C (NPC) at the Children's Hospital & Research Center Oakland.
Biotechnology company CTD Holdings Inc (Other OTC:CTDH) revealed on Tuesday that it has received US Food and Drug Administration (FDA) Fast Track designation for Trappsol Cyclo for the treatment of the rare and fatal genetic disease Niemann-Pick Disease Type C (NPC).
Biotechnology company CTD Holdings (Other OTC:CTDH) disclosed on Wednesday that it has submitted a Clinical Trial Application (CTA) in Italy to launch a Phase I/II clinical trial of its Trappsol Cyclo for the treatment of Niemann-Pick Disease Type C.
M2 PHARMA-December 22, 2016-CTD Holdings files Clinical Trial Application in Italy to initiate Phase I/II clinical trial of Trappsol Cyclo for Niemann-Pick Disease Type C
Three-year-old Lucy Mathieson has the terminal Niemann-Pick disease type C, which affects just over 300 people worldwide.