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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

20.02.20

Cyclo Therapeutics Announces Completion of Enrollment in its Phase I/II Trial to Evaluate Trappsol® Cyclo™ for the Treatment of Niemann-Pick Disease Type C

Cyclo Therapeutics, Inc. (OTCQB: CTDH), a clinical-stage biotechnology company that develops cyclodextrin-based products for the treatment of Niemann-Pick Disease Type C and Alzheimer’s Disease, today announced that it has completed patient enrollment in its Phase I/II trial to evaluate the safety, tolerability, and efficacy of Trappsol® Cyclo™ administered intravenously to Niemann-Pick Disease Type C1 (NPC1) patients...

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12.02.20

Last Patient Last Visit in Cyclo Therapeutics’ Phase I Trial in Niemann-Pick Type C

GAINESVILLE, FL – (Businesswire) – February 11, 2020 – Cyclo Therapeutics, Inc. (OTCQB: CTDH), a biotechnology company that develops cyclodextrin-based products for the treatment of Niemann-Pick Disease Type C (NPC) and Alzheimer’s Disease, today announced that the last patient has undergone the final assessment in its Phase I trial to evaluate the safety and tolerability of Trappsol® Cyclo™ administered intravenously to NPC patients.

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