FDA Approves First Targeted Treatment for Cerebrotendinous Xanthomatosis: A Perspective on a Landmark in Rare Lipid Storage Disease Therapy
The FDA approval of chenodiol marks a significant milestone in the management of CTX, providing a standardized and evidence-based therapy for a previously neglected condition. Early diagnosis, broader screening, and global access to the drug will be key to improving outcomes and ensuring lasting clinical benefit.
Authors
- Areeba Aamir Ali Basaria (ORCID: https://orcid.org/0000-0003-1087-3836)
- Laiba Jalal (ORCID: https://orcid.org/0009-0003-4147-3138)
- Hermann Yokolo (ORCID: https://orcid.org/0009-0002-8347-7236)
Institutions
- Dow University of Health Sciences (PK)
Publication Details
- Journal
- Health Science Reports
- Published
- 2025-11-26
- DOI
- https://doi.org/10.1002/hsr2.71549
- Citations
- 1
- Primary Topic
- Cholesterol and Lipid Metabolism
- Type
- article
- Field-Weighted Citation Impact
- 2.01