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We aim to provide up-to-date, comprehensive and practical information and advice for those affected by Niemann-Pick diseases as well as information and resources specifically for health, education and social care professionals. The information we develop covers a broad range of topics to enable you to tailor information for your needs. Please feel free to share any of the resources available. If you have any questions or need further information then please contact us at any time.

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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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