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FDA Breakthrough: First-Ever Treatment Approved for Rare and Devastating CTX Disorder!
Administered at a dose of 250 milligrams three times daily, the treatment demonstrated significant reductions in plasma cholestanol and urinary 23S-pentol—key biomarkers elevated in CTX patients.
WebMD29d
FDA Approves New Treatment for Rare Lipid Storage Disease
The FDA advises 250 mg tablets to be taken thrice a day. Ctexli is the first approved drug for the condition. A 24-week clinical trial tested how well Ctexli worked in people with CTX ages 16 to ...
Yahoo Finance28d
FDA approves Mirum’s Ctexli for cerebrotendinous xanthomatosis
Subjects treated with Ctexli, at a dose of 250 mg three times per day ... metabolic diseases like cerebrotendinous xanthomatosis. “CTX is a progressive multisystemic disorder that significantly ...
Benzinga.com1mon
1MIRM : FDA Approves Mirum Pharmaceuticals' Drug As First For Genetic Disorder...
250 milligrams three times per day, significantly reduced plasma cholestanol and urine 23S-pentol (cholesterol metabolites markedly increased in CTX patients) compared to placebo treatment.
Healio1mon
FDA approves first treatment for rare lipid storage disease
“Today’s approval provides a safe and effective treatment option for CTX.” The double-blind, placebo-controlled randomized trial demonstrated 250 mL Ctexli three times per day orally ...
Longview News-Journal1mon
FDA Approves First Treatment for Cerebrotendinous Xanthomatosis, a Rare Lipid Storage Disease
250 milligrams three times per day, resulted in significant reduction in plasma cholestanol and urine 23S-pentol (cholesterol metabolites that are markedly increased in CTX patients) compared to ...