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Mirum Pharmaceuticals, a rare disease company, expects $500mln-$510mln in revenues this year and is cash flow positive. The company has a full pipeline led by volixibat for adult cholestatic diseases and MRM-3379 for Fragile X syndrome, where the first patient was enrolled in a Phase II trial yesterday. Mirum aims to tap into the $1 bln-plus Fragile X market.

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