Raw research findings for this medication. Mostly single studies, not curated or confirmed robust.
Ataluren is designed to let the cell's ribosomes read through a premature stop signal (a nonsense mutation) in a gene, so a full-length protein can still be made. It was conditionally approved in the EU for Duchenne muscular dystrophy caused by a nonsense mutation in the dystrophin gene, and studied in cystic fibrosis; the EU approval was later not renewed. It was sold as Translarna.
Ataluren is relevant only for people whose condition is caused by a nonsense mutation, in DMD (dystrophin) for Duchenne muscular dystrophy or in CFTR for cystic fibrosis. The studies on this page concern CFTR nonsense mutations in cystic fibrosis trials, where it did not show a clear benefit overall. There is no CPIC or DPWG guideline.
Every published finding on gene variants and Ataluren. 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 rs77010898: Level 3. Other: Cystic Fibrosis. 4.
CFTR rs113993959: Level 3. Other: Cystic Fibrosis. 4.
CFTR rs75039782: Level 3. Other: Cystic Fibrosis. 2.
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
Aldara (Immune Response Modifiers)
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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