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  1. MIPLYFFA® and Zevra Therapeutics presented new clinical data on a drug targeting the underlying pathology of Niemann-Pick disease type C at the International Congress of Inborn Errors of Metabolism.

The FDA approved Miplyffa for the treatment of Niemann-Pick disease.

1 report, 0 independent Updated Jul 3
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The FDA approved Miplyffa for the treatment of Niemann-Pick disease.

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