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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

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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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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