Raw research findings for this medication. Mostly single studies, not curated or confirmed robust.
Cysteamine is a cystine-depleting medicine for cystinosis, a rare inherited condition in which the amino acid cystine builds up inside cells and damages the kidneys, eyes and other organs. It reacts with cystine so it can leave the cells. It is taken as capsules (Cystagon, Procysbi) and as eye drops (Cystaran, Cystadrops).
Cysteamine treats cystinosis caused by variants in CTNS, the gene for the lysosomal cystine transporter, regardless of the specific variant. The gene study listed came from research testing cysteamine, combined with other compounds, in cystic fibrosis with the F508del CFTR variant. There is no CPIC or DPWG guideline.
Every published finding on gene variants and Cysteamine. Upload your DNA file to see which of these rows apply to you.
PharmGKB weighs all studies of a variant and rates how strong the evidence is, from 1A (strongest) to 4.
CFTR rs113993960: Level 3. Efficacy: Cystic Fibrosis. 1.
Studies of how well it worked in people carrying a variant.
Study findings and evidence levels: PharmGKB (CC BY-SA 4.0). Guidelines: CPIC and DPWG, as bundled in PharmCAT. Most studies were done mainly in people of European ancestry.
More drugs from the same category
Antithyroid Drugs (class) (Antithyroid Agents)
Not a prescribing recommendation. This page is educational information about pharmacogenomic research and clinical guidelines - it is not medical or dosing advice, and not a prediction of how any medication will work for you. Never start, stop, or change a medication or dose based on it; talk to your doctor or pharmacist, who can order clinical-grade testing if appropriate.
Content updated September 2026.
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