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  1. 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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  2. 13.09.19

    26th Annual Family Conference & Interactive Workshop on Niemann-Pick Diseases 2019 (PROGRAMME)

    We are pleased to share the programme for the upcoming 26th Annual Family Conference & 10th Interactive Workshop on Niemann-Pick Diseases. We look forward to seeing our community all together once again, if you have any issues or queries regarding this event please do not hesitate to get in touch with us by either email at: or phone on: 0191 415 06 93.

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  3. 12.09.19

    CTD Holdings to Present at 2019 Annual Conference for NPC Patients, Families, and Health Professionals in the United Kingdom

    CTD is also a proud sponsor of the NPUK Annual Family Conference & Interactive Workshop on Niemann-Pick Diseases, for the 4th year running...

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  4. 12.09.19

    ASMD Research Study Opportunity

    Evidera, a research group working on behalf of Sanofi Genzyme, is conducting a research study to understand the symptoms and impact experienced by carers of those affected by ASMD Niemann-Pick disease types B, or A/B...

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  5. 30.08.19


    Copenhagen, Denmark, August 28, 2019 – Orphazyme A/S (ticker: ORPHA.CO), a biopharmaceutical company dedicated to developing treatments for patients living with rare diseases, today announces its interim financial results for the period January 1 – June 30, 2019 and corporate highlights...

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