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    • Pompe disease

    - PMLiVE

    Shionogi gains rights to Maze’s investigational Pompe disease therapy for $150m upfront

    The inherited disorder is estimated to affect about one in 40,000 people in the US

    - PMLiVE

    Sanofi presents positive results for Pompe disease enzyme replacement therapy

    The rare genetic disorder is estimated to affect around one in every 40,000 people in the UK

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    European Commission approves Amicus Therapeutics’ Pompe disease therapy

    The company can now offer Opfolda together with its previously-approved Pombiliti

    - PMLiVE

    Sanofi signs agreement worth up to $750m for Maze’s Pompe disease drug

    The candidate has recently cleared its first phase 1 clinical trial

    - PMLiVE

    Sanofi’s enzyme replacement therapy available on NHS to Pompe disease patients

    The rare genetic disorder affects around one in every 40,000 people in the UK

    - PMLiVE

    European Commission approves potential new treatment for Pompe disease

    Nexviadyme is the first and only newly approved medicine for Pompe disease in Europe in over 15 years

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