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- FDA launches rare disease framework initiativeA new initiative from the US Food and Drug Administration (FDA) aims to develop “an optimized, fit-for-purpose” framework to facilitate the development and marketing of treatments for rare diseases…raps · 5d ago↗ source
- FDA approves Egetis’ Emcitate for rare genetic disease as Novo secures Chinese weight-loss pill in $2.6 billion dealThe U.S. Food and Drug Administration has granted approval to Egetis' Emcitate, a therapy targeting a rare genetic disorder. This marks a regulatory milestone for the company and patients awaiting…