REGULATORY

Japan Doles Out Orphan Tags to Ultragenyx’s GTX-102 and Lots More

September 1, 2025
Japan’s Ministry of Health, Labor and Welfare (MHLW) on August 29 granted orphan drug designation to a long list of new and existing compounds including Ultragenyx Japan’s GTX-102 for the proposed indication of Angelman syndrome. The designated compounds and expected…

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REGULATORY

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