The FDA has approved Ctexli for the treatment of cerebrotendinous xanthomatosis (CTX), a rare lipid storage disease, in adults. According to a press release, Ctexli (chenodiol) is the first FDA ...

A combination of Drosophila dGBA1b loss-of-function models and Gaucher Disease (GD) patient-derived iPSCs reveals an impairment in GD neuronal cell growth and that Hippo pathway hyperactivation ...

Mirum Pharma's Ctexli has become the first FDA-approved therapy for cerebrotendinous xanthomatosis (CTX), an ultra-rare form of lipid storage disease. Ctexli (chenodiol) has been cleared to treat ...

People at higher risk for heart disease may also be more likely to develop dementia, according to new research led by The ...

Feb. 24, 2025 – The FDA has approved a new pill for adults with cerebrotendinous xanthomatosis (CTX), a rare genetic disease that makes it hard for the body to handle cholesterol properly.

Ctexli is the first FDA-approved drug to treat CTX, a very rare lipid storage disease. "The FDA is dedicated to supporting new drug development for rare diseases including very rare metabolic ...

Ctexli is the first FDA-approved drug to treat CTX, a very rare lipid storage disease. "The FDA is dedicated to supporting new drug development for rare diseases including very rare metabolic diseases ...