{"id":"srp-4053","safety":{"commonSideEffects":[{"rate":null,"effect":"Injection site reactions"},{"rate":null,"effect":"Immune-mediated toxicity"},{"rate":null,"effect":"Glomerulonephritis"}]},"_chembl":{"chemblId":"CHEMBL604529","moleculeType":"Small molecule","molecularWeight":"548.04"},"_dailymed":null,"mechanism":{"_ai_source":"anthropic-haiku","explanation":"SRP-4053 binds to exon 53 of the dystrophin pre-mRNA and causes the splicing machinery to skip this exon during mRNA processing. This allows the production of a shorter but functional dystrophin protein (micro-dystrophin), which can partially restore muscle function in patients with specific DMD mutations. The drug is designed for patients whose mutations can be corrected by exon 53 skipping.","oneSentence":"SRP-4053 is an antisense oligonucleotide that modulates dystrophin production by promoting exon 53 skipping in Duchenne muscular dystrophy patients with mutations amenable to this approach.","_ai_confidence":"medium"},"_scrapedAt":"2026-03-28T01:36:01.699Z","_scrapedBy":"cloudflare-swarm","_wikipedia":null,"indications":{"approved":[{"name":"Duchenne muscular dystrophy (DMD) with mutations amenable to exon 53 skipping"}]},"trialDetails":[{"nctId":"NCT02500381","phase":"PHASE3","title":"Study of SRP-4045 (Casimersen) and SRP-4053 (Golodirsen) in Participants With Duchenne Muscular Dystrophy (DMD)","status":"COMPLETED","sponsor":"Sarepta Therapeutics, Inc.","startDate":"2016-09-28","conditions":"Duchenne Muscular Dystrophy","enrollment":228},{"nctId":"NCT06606340","phase":"","title":"A Long-term Observational Study Evaluating Eteplirsen, Golodirsen, or Casimersen in Routine Clinical Practice","status":"ENROLLING_BY_INVITATION","sponsor":"Sarepta Therapeutics, Inc.","startDate":"2019-01-07","conditions":"Duchenne Muscular Dystrophy","enrollment":300},{"nctId":"NCT03532542","phase":"PHASE3","title":"An Extension Study to Evaluate Casimersen or Golodirsen in Patients With Duchenne Muscular Dystrophy","status":"TERMINATED","sponsor":"Sarepta Therapeutics, Inc.","startDate":"2018-08-02","conditions":"Duchenne Muscular Dystrophy","enrollment":171},{"nctId":"NCT04179409","phase":"PHASE2","title":"A 48-Week, Open Label, Study to Evaluate the Efficacy and Safety of AMONDYS 45, EXONDYS 51, VYONDYS 53 in Subjects With DuchenneMuscular Dystrophy Carrying Eligible DMD Duplications.","status":"COMPLETED","sponsor":"Kevin Flanigan","startDate":"2020-02-18","conditions":"Duchenne Muscular Dystrophy","enrollment":3},{"nctId":"NCT02310906","phase":"PHASE1, PHASE2","title":"Phase I/II Study of SRP-4053 in DMD Patients","status":"COMPLETED","sponsor":"Sarepta Therapeutics, Inc.","startDate":"2015-01-13","conditions":"Duchenne Muscular Dystrophy","enrollment":39}],"_emaApprovals":[],"_faersSignals":[],"_approvalHistory":[],"publicationCount":6,"rwe":[],"genericFilers":[],"relatedDrugs":[],"labelChanges":[],"biosimilarFilings":[],"pricing":[],"formularyStatus":[],"manufacturing":[],"companionDiagnostics":[],"competitors":[],"timeline":[],"patents":[],"ownershipHistory":[],"trials":[],"biosimilars":[],"latestUpdates":[],"references":[],"tags":[],"ecosystem":[],"genericManufacturerList":[],"offLabel":[],"developmentCodes":[],"aliases":["Golodirsen","VYONDYS 53"],"phase":"phase_3","status":"active","brandName":"SRP-4053","genericName":"SRP-4053","companyName":"Sarepta Therapeutics, Inc.","companyId":"sarepta-therapeutics-inc","modality":"Small molecule","firstApprovalDate":"","aiSummary":"SRP-4053 is an antisense oligonucleotide that modulates dystrophin production by promoting exon 53 skipping in Duchenne muscular dystrophy patients with mutations amenable to this approach. Used for Duchenne muscular dystrophy (DMD) with mutations amenable to exon 53 skipping.","enrichmentLevel":3,"visitCount":1,"trialStats":{"total":2,"withResults":1},"verificationStatus":"verified","dataCompleteness":{"mechanism":true,"indications":true,"safety":true,"trials":true,"score":4}}