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ERT

This page shows the latest ERT news and features for those working in and with pharma, biotech and healthcare.

European Commission approves potential new treatment for Pompe disease

European Commission approves potential new treatment for Pompe disease

muscles. Nexviadyme is an enzyme replacement therapy (ERT) and is currently the first and only newly approved medicine for Pompe disease in Europe in over 15 years. ... Nexviadyme is specifically designed to target the mannose-6-phosphate (M6P) receptor,

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  • Rare diseases: not so rare after all Rare diseases: not so rare after all

    Identification of the specific enzyme involved, which is either defective or absent, has led to the development of a first-generation treatment option: enzyme replacement therapy (ERT). ... ERT provides the patient with the recombinantly produced missing

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