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Niemann-Pick Disease

Niemann-Pick diseases are a group of rare and devastating inherited lysosomal storage disorders that can affect both children and adults:

Acid Sphingomyelinase Deficiency (ASMD) includes Niemann-Pick Disease type A (NP-A) and type B (NP-B), which are caused by a lack of the enzyme acid sphingomyelinase leading to a build-up of toxic materials in the body. Niemann-Pick Disease Type C (NP-C) is a hugely life-limiting neurodegenerative disease caused by an accumulation of lipids (fats) in the liver, brain and spleen.

At NPUK we are dedicated to supporting the individuals and families affected by these conditions. We offer practical and emotional support, provide information and strive to raise further awareness of the disease(s) in the hope of facilitating research and potential therapies.

Niemann-Pick Disease

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Latest news

15.03.19

CTD Holdings Announces Plan to Launch Clinical Trial of Trappsol® Cyclo™ in Alzheimer’s Disease

ALACHUA, FL – (Globe Newswire) – March 14, 2019 – CTD Holdings, Inc. (OTCQB: CTDH), a clinical stage biotechnology company that develops cyclodextrin-based products for the treatment of disease with unmet medical need, announced today that the company has initiated planning for a Phase I clinical trial for treatment of Alzheimer’s Disease. The trial will use Trappsol® Cyclo™, the company’s proprietary formulation of hydroxypropyl beta cyclodextrin, administered intravenously.

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11.03.19

IntraBio Clinical Trial Authorization Application Approved by the MHRA for the Treatment of Niemann-Pick Disease type C (NPC)

IntraBio Clinical Trial Authorization Application Approved by the MHRA for the Treatment of Niemann-Pick Disease type C (NPC): OXFORD, UK, March 11, 2019-- IntraBio Inc., a late-stage biopharmaceutical company, today announced that the UK Medicines and Healthcare products Regulatory Agency (MHRA) has approved its Clinical Trial Authorisation (CTA) Application for Clinical Trial IB1001-201 with its lead compound (IB1001) for the treatment of Niemann-Pick disease Type C (NPC).

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