FDA Approves Mirum Pharmaceuticals' Drug As First For Genetic Disorder With Inability To Break-Down Cholesterol

FDA Approves Mirum Pharmaceuticals' Drug As First For Genetic Disorder With Inability To Break-Down Cholesterol

Submitted by admin on February 23, 2025 - 10:38pm
Source: 
Benzinga
News Tags: 
Mirum Pharmaceuticals
FDA
Ctexli
cerebrotendinous xanthomatosis
Headline: 
FDA Approves Mirum Pharmaceuticals' Drug As First For Genetic Disorder With Inability To Break-Down Cholesterol
snippet: 
  • FDA approves Mirum's Ctexli, the first treatment for cerebrotendinous xanthomatosis, a rare genetic disorder affecting fat metabolism.
  • In a trial, Ctexli significantly reduced cholesterol metabolites linked to CTX, but it carries a liver toxicity warning requiring monitoring

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