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- FDA approves Mirum's Atebrioz for ultra-rare disease that hardens muscle and connective tissueThe U.S. Food and Drug Administration has granted approval for Mirum Pharmaceuticals' new therapy for an ultra-rare genetic disorder that causes muscle and connective tissue to turn into bone. The…
- Experts say expanding beyond rare disease markets can make ultra-rare therapies financially viableThe piece highlights that investment in ultra‑rare disease treatments remains limited, and experts argue the answer is not to focus solely on the rare condition itself. They propose that drugmaker…