CTX stems from a genetic mutation in the CYP27A1 gene, resulting in a deficiency of an enzyme essential for lipid breakdown. This deficiency disrupts bile acid production in the liver, preventing ...

According to the Technical Service Bulletin (TSB) N222389722 issued on August 28, 2024, GM acknowledged the condition where ...

OHSU's pioneering research, clinical trials led to better testing, approval for CTX disease Eric Stewart has a rare disease ...

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.

Cingulate Inc. submitted CTx-1301 Phase 3 safety data to the FDA, preparing for a Pre-NDA meeting on April 2, 2025. Cingulate Inc. announced the submission of Phase 3 safety data for its ADHD ...

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 ...

The approval is based on data from the Phase 3 RESTORE study evaluating the safety and efficacy of CTEXLI in adult patients with CTX by measurement of urine bile alcohols and other secondary measures.

Top-line Phase 2/3 data readout for COMPANION-002, evaluating tovecimig (CTX-009 - a DLL4 x VEGF-A bispecific antibody) in patients with biliary tract cancer (BTC), is on track for the end of the ...

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