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Without people like you getting involved as a volunteer, fundraiser, or anything in between - we wouldn't be able to provide the care and support that we provide to families affected by Niemann-Pick disease! There are numerous opportunities available when it comes to helping us - the only limit is your imagination; there's everything from taking part in our fun days as a member of our passionate volunteer team, setting up fundraisers, or drumming up interest and donations through creative sponsored events. By working together we can make a real difference to the lives of those people affected by Niemann-Pick disease. Please contact us for more information on how you can join us, by email at: info@npuk.org or by phone on: 0191 415 06 93.

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Latest news

13.09.19

CTD Holdings Announces FDA Approval of US Expanded Access Treatment Program in Niemann-Pick Disease Type C

ALACHUA, FL–(Businesswire)–13 September 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, today announced that the company will provide Trappsol® Cyclo™, its proprietary hydroxypropyl beta cyclodextrin drug, to a pediatric patient diagnosed with Niemann-Pick Disease Type C...

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13.09.19

IntraBio: Clinical Trial IB1001-201: US Recruitment

IB1001-201 is a multinational study that investigates the effects of the novel drug IB1001 (N-Acetyl-LLeucine) for treatment of Niemann-Pick Disease type C (NPC). The trial has previously been accepted by Independent Ethics Committees (IEC), and the UK Medicines and Healthcare products Regulatory Agency (EudraCT: 2018-004431-71), as well as National Regulatory Agencies in Germany, Slovakia, and Spain, and the US Food and Drug Administration (IND 134369)...

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