Cerebrotendinous xanthomatosis
Also known as CTX, CTx, cholestanol storage disease, sterol 27-hydroxylase deficiency+1 more
CTX, CTx, cholestanol storage disease, sterol 27-hydroxylase deficiency, cerebral cholesterinosis.
What's happening now
An analyst briefing on current research, clinical, regulatory and industry activity surrounding this disease.
Therapeutic landscape
Therapies with a regulatory footing for this condition, alongside the wider set of agents co-studied with it in the literature.
Approval — Chenodeoxycholic acid is indicated for the treatment of inborn errors of primary bile aci… (2017)
Clinical trials
The current development programme across all trial phases.
Regulatory timeline
Drug regulatory events matched to this condition by indication — EMA.
European Medicines Agency (CC BY 4.0). Events are matched to this condition by drug indication text — approvals/updates for drugs indicated for it, not disease-specific acts.
Associated genes
Genes associated with this disease in the canonical knowledge graph (Open Targets evidence). Number shows the association score.
Disease biology
Proteins whose encoding gene is associated with this disease, reached through the canonical gene→disease graph. Number shows the gene's association score.
Reference
Authoritative identity, definition & identifiers.
CTX, CTx, cholestanol storage disease, sterol 27-hydroxylase deficiency, cerebral cholesterinosis
- Disease identity & definition — NLM Medical Subject Headings (MeSH), public domain
- Clinical trials — ClinicalTrials.gov (U.S. National Library of Medicine)
- Related entities are derived from literature co-mention (studied together) — associative, not causal.