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FDA Approves First Drug for Rare Lipid Storage Disorder

Summary by MedPage Today
(MedPage Today) -- The FDA on Friday approved chenodiol (Ctexli) for the treatment of cerebrotendinous xanthomatosis (CTX) in adults. Chenodiol -- a naturally occurring human bile acid also known as chenodeoxycholic acid -- is the first FDA...

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MedPage TodayMedPage Today

FDA Approves First Drug for Rare Lipid Storage Disorder

(MedPage Today) -- The FDA on Friday approved chenodiol (Ctexli) for the treatment of cerebrotendinous xanthomatosis (CTX) in adults. Chenodiol -- a naturally occurring human bile acid also known as chenodeoxycholic acid -- is the first FDA...

·New York, United States
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Seeking AlphaSeeking Alpha

Mirum gains FDA approval of chenodiol for rare lipid storage disease

FDA approves Mirum's Ctexli, the first treatment for CTX, a rare genetic disorder. Read more here.

·United States
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The Kane RepublicanThe Kane Republican
+11 Reposted by 11 other sources

FDA Approves First Treatment for Cerebrotendinous Xanthomatosis, a Rare Lipid Storage Disease

SILVER SPRING, Md., Feb. 21, 2025 /PRNewswire/ -- Today, the U.S. Food and Drug Administration approved Ctexli (chenodiol) for the treatment of cerebrotendinous xanthomatosis (CTX) in adults. Ctexli is the first FDA-approved drug to treat CTX, a very rare lipid…

·Kane, United States
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drugtopics.comdrugtopics.com

FDA Approves Chenodiol for Treatment of Cerebrotendinous Xanthomatosis

Ctexli is the first FDA-approved treatment for adults with cerebrotendinous xanthomatosis.

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Pharmacy TimesPharmacy Times

Chenodiol, First Treatment for Cerebrotendinous Xanthomatosis, Receives FDA Approval

This marks the first treatment to be FDA-approved for this rare lipid storage disease.

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hcplive.comhcplive.com

FDA Approves Chenodiol Tablets for Cerebrotendinous Xanthomatosis

With this FDA approval, chenodiol tablets become the first approved drug to treat CTX, a rare lipid storage disease.

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lavozdelsur.es broke the news in on Friday, February 21, 2025.
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