All transthyretin (ATTR) amyloidosis with cardiomyopathy articles
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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.
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NewsRegulatory-first for gene editing therapy
The world’s first investigational in vivo CRISPR-based gene editing therapy cleared for late-stage clinical development is expected to enter Phase III in late 2023.