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  1. 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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  2. 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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  3. 11.08.19

    Questions from the NPC community regarding the suspension of VTS-270 301 in UK and France (Mallinckrodt)

    Mallinckrodt answer questions from the NPC community regarding the suspension of VTS-270 301 in UK and France...

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  4. 02.08.19

    Mallinckrodt: official statement regarding the Study 301 suspension

    The following article features a letter regarding the Study 301 Suspension from Sheila Talafous, Director of Advocacy Relations at Mallinckrodt Pharmaceuticals...

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  5. 26.07.19

    Survey Opportunity: Qualitative Analysis of Niemann-Pick Type C 5-Domain NPCCSS

    We are pleased to inform you of a study opportunity available to NPC patients and their families:
    Clinical Outcomes Solutions (COS), is a research company conducting research into Niemann-Pick type C (NPC) on behalf of a sponsor, Orphazyme A/S, a pharmaceutical company studying NPC...

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