{"id":"ataluren","rwe":[],"tags":[{"label":"Small Molecule","category":"modality"},{"label":"Flavin reductase (NADPH)","category":"target"},{"label":"BLVRB","category":"gene"},{"label":"M09AX03","category":"atc"},{"label":"Active","category":"status"},{"label":"Duchenne muscular dystrophy","category":"indication"},{"label":"PTC Therapeutics International Limited","category":"company"}],"phase":"marketed","safety":{"commonSideEffects":[]},"trials":[],"aliases":[],"company":"PTC Therapeutics International Limited","patents":[],"pricing":[],"_sources":{"trials":{"url":"https://clinicaltrials.gov/search?intr=ATALUREN","method":"api_direct","source":"ClinicalTrials.gov","rawText":"","confidence":1,"sourceType":"ctgov","retrievedAt":"2026-04-20T03:33:44.402059+00:00"},"regulatory.ca":{"url":"","method":"api_direct","source":"Health Canada DPD","rawText":"","confidence":1,"sourceType":"health_canada_dpd","retrievedAt":"2026-04-20T03:33:49.722210+00:00"},"regulatory.eu":{"url":"","method":"api_direct","source":"European Medicines Agency","rawText":"","confidence":1,"sourceType":"ema_api","retrievedAt":"2026-04-20T03:33:44.475555+00:00"},"publicationCount":{"url":"https://pubmed.ncbi.nlm.nih.gov/?term=ATALUREN","method":"api_direct","source":"PubMed/NCBI","rawText":"","confidence":1,"sourceType":"pubmed","retrievedAt":"2026-04-20T03:33:50.162371+00:00"},"mechanism.target_chembl":{"url":"","method":"api_direct","source":"ChEMBL mechanism: 80S Ribosome modulator","rawText":"","confidence":1,"sourceType":"chembl","retrievedAt":"2026-04-20T03:33:51.213619+00:00"},"crossReferences.chemblId":{"url":"https://www.ebi.ac.uk/chembl/compound_report_card/CHEMBL256997/","method":"api_direct","source":"ChEMBL (EMBL-EBI)","rawText":"","confidence":1,"sourceType":"chembl","retrievedAt":"2026-04-20T03:33:50.864076+00:00"}},"allNames":"translarna","offLabel":[],"synonyms":["ataluren","translarna","PTC124"],"timeline":[{"date":"2014-07-31","type":"positive","source":"DrugCentral","milestone":"EMA approval (PTC Therapeutics International Limited)"}],"aiSummary":"Translarna (ataluren) is a small molecule modality developed by PTC Therapeutics International Limited, targeting flavin reductase (NADPH) to treat Duchenne muscular dystrophy. It is currently owned by PTC Therapeutics International Limited and has been approved for its indication. The commercial status of Translarna is patented, and its safety considerations include potential liver enzyme elevations and gastrointestinal side effects. Translarna works by bypassing premature stop codons in the genetic code, allowing for the production of functional dystrophin protein. Further clinical data and long-term studies are needed to fully understand its efficacy and safety profile.","brandName":"Translarna","ecosystem":[{"indication":"Duchenne muscular dystrophy","otherDrugs":[{"name":"casimersen","slug":"casimersen","company":"Sarepta Theraps Inc"},{"name":"deflazacort","slug":"deflazacort","company":"Marathon Pharmaceuticals Llc"},{"name":"eteplirsen","slug":"eteplirsen","company":"Sarepta Therapeutics Inc"},{"name":"golodirsen","slug":"golodirsen","company":"Sarepta Theraps Inc"}],"globalPrevalence":224000}],"mechanism":{"target":"Flavin reductase (NADPH)","targets":[{"gene":"BLVRB","source":"DrugCentral","target":"Flavin reductase (NADPH)","protein":"Flavin reductase (NADPH)"}],"modality":"Small Molecule","explanation":"","oneSentence":"","technicalDetail":"Translarna is a small molecule that targets the flavin reductase (NADPH) enzyme, which is involved in the nonsense-mediated mRNA decay pathway. By inhibiting this enzyme, Translarna allows for the translation of mRNA with premature stop codons, leading to the production of functional dystrophin protein."},"commercial":{},"references":[{"id":1,"url":"https://drugcentral.org/drugcard/4872","fields":["approvals","synonyms","ATC","PK","indications","contraindications","DDIs","targets","patents","FAERS"],"source":"DrugCentral"},{"id":2,"url":"https://clinicaltrials.gov/search?intr=ATALUREN","fields":["trials"],"source":"ClinicalTrials.gov"},{"id":3,"url":"https://pubmed.ncbi.nlm.nih.gov/?term=ATALUREN","fields":["publications"],"source":"PubMed/NCBI"}],"_emaChecked":true,"_enrichedAt":"2026-03-30T08:50:37.113770","_validation":{"fieldsValidated":0,"lastValidatedAt":"2026-04-20T03:33:52.438615+00:00","fieldsConflicting":1,"overallConfidence":0.8},"biosimilars":[],"competitors":[{"drugName":"hyaluronic 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