All sepiapterin articles
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News
Broad label FDA approval for phenylketonuria drug
Alongside Phase III data demonstrating significant efficacy in the rare disease, the FDA’s decision could position the small molecule as a future standard of care.
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NewsCHMP meeting highlights – April 2025
The Committee for Medicinal Products for Human Use (CHMP) recommended approval of a variety of new medicines, including several treatments for hereditary and rare conditions.