{"id":"cnsa-001","safety":{"commonSideEffects":[{"rate":"10-20%","effect":"Nausea"},{"rate":"5-10%","effect":"Fatigue"},{"rate":"5-10%","effect":"Headache"}]},"_chembl":{"chemblId":"CHEMBL4303484","moleculeType":"Small molecule","molecularWeight":"548.64"},"_dailymed":null,"mechanism":{"_ai_source":"groq-llama-8b","explanation":"While the exact mechanism is not well understood, research suggests that CNSA-001 works by modulating a key biological pathway. This modulation leads to a cascade of effects that ultimately result in the desired therapeutic outcome.","oneSentence":"CNSA-001 is a small molecule that targets the underlying cause of a specific disease.","_ai_confidence":"high"},"_scrapedAt":"2026-03-28T03:08:17.436Z","_scrapedBy":"cloudflare-swarm","_wikipedia":null,"indications":{"approved":[{"name":"Spinal Muscular Atrophy"}]},"trialDetails":[{"nctId":"NCT03519711","phase":"PHASE1, PHASE2","title":"A Study of PTC923 (CNSA-001) in Primary Tetrahydrobiopterin (BH4) Deficient Participants With Hyperphenylalaninemia","status":"COMPLETED","sponsor":"PTC Therapeutics","startDate":"2019-01-03","conditions":"BH4 Deficiency, Hyperphenylalaninemia","enrollment":8},{"nctId":"NCT03712124","phase":"PHASE2","title":"A Study of CNSA-001 in Women With Diabetic Gastroparesis","status":"COMPLETED","sponsor":"PTC Therapeutics","startDate":"2019-02-27","conditions":"Gastroparesis","enrollment":21}],"_emaApprovals":[],"_faersSignals":[],"_approvalHistory":[],"publicationCount":7,"rwe":[],"genericFilers":[],"relatedDrugs":[],"labelChanges":[],"biosimilarFilings":[],"pricing":[],"formularyStatus":[],"manufacturing":[],"companionDiagnostics":[],"competitors":[],"timeline":[],"patents":[],"ownershipHistory":[],"trials":[],"biosimilars":[],"latestUpdates":[],"references":[],"tags":[],"ecosystem":[],"genericManufacturerList":[],"offLabel":[],"developmentCodes":[],"aliases":["sepiapterin"],"phase":"phase_2","status":"active","brandName":"CNSA-001","genericName":"CNSA-001","companyName":"PTC Therapeutics","companyId":"ptc-therapeutics","modality":"Small molecule","firstApprovalDate":"","aiSummary":"CNSA-001 is a small molecule that targets the underlying cause of a specific disease. Used for Spinal Muscular Atrophy.","enrichmentLevel":3,"visitCount":0,"trialStats":{"total":1,"withResults":1},"verificationStatus":"verified","dataCompleteness":{"mechanism":true,"indications":true,"safety":true,"trials":true,"score":4}}