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  1. 27.03.20

    A message from Orphazyme to the global community of patients, family members and healthcare providers impacted by the corona virus pandemic (COVID-19):

    In the days and weeks to come, Orphazyme will continue to communicate openly with patient organizations and clinical trial investigators around the globe to share the latest information regarding our efforts as the situation develops. Meanwhile please utilize attached FAQs to answer questions if any about our trials...

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  2. 25.03.20

    IntraBio Receives FDA Fast Track Designation for Niemann-Pick Disease

    OXFORD, UK / ACCESSWIRE / March 25, 2020 / IntraBio Inc., a late-stage biopharmaceutical company, today announced that the US Food and Drug Administration (FDA) has granted Fast Track designation to its lead compound (IB1001) for the treatment of Niemann-Pick disease Type C (NPC)...

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  3. 25.03.20

    Cyclo Therapeutics Inc. Announces Positive Safety Profile of its Drug Trappsol® Cyclo™ in the Treatment of Niemann Pick Disease Type C

    GAINESVILLE, FL – (Businesswire) – March 23, 2020 – Cyclo Therapeutics, Inc. (OTCQB: CTDH), a clinical-stage 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 Safety Review Committee for its Phase I trial in NPC has determined that Trappsol® Cyclo™ has an acceptable safety profile in patients with Niemann-Pick Disease Type C1...

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  4. 23.03.20

    COVID-19 Webinar, Monday 23rd March

    This webinar has been organised by patient organisations working in the field of Inherited Metabolic Diseases (IMD). It is open to patients and families affected by IMD and it aims to address questions raised by the outbreak of novel coronavirus (COVID-19)...

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  5. 20.03.20

    Letter from our Chief Executive: Toni Mathieson

    Toni Mathieson, NPUK Chief Executive: I wanted to take this opportunity to let our patients, families and supporters know how NPUK is responding to the coronavirus (COVID-19) pandemic...

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