{"id":"placebo-rhasb","safety":{"commonSideEffects":[{"rate":null,"effect":"Infusion-related reactions"},{"rate":null,"effect":"Antibody formation"},{"rate":null,"effect":"Pyrexia"},{"rate":null,"effect":"Headache"}]},"_chembl":null,"_dailymed":null,"mechanism":{"_ai_source":"claude-haiku-4.5","explanation":"Maroteaux-Lamy syndrome (mucopolysaccharidosis VI) is caused by deficiency of the enzyme arylsulfatase B, leading to accumulation of dermatan sulfate in tissues. rhASB is a recombinant form of this enzyme that catalyzes the breakdown of accumulated glycosaminoglycans, reducing tissue burden and improving organ function. The enzyme is administered intravenously to reach systemic circulation and target affected tissues.","oneSentence":"rhASB (recombinant human arylsulfatase B) is an enzyme replacement therapy that replaces deficient arylsulfatase B to break down glycosaminoglycans accumulated in Maroteaux-Lamy syndrome.","_ai_confidence":"medium"},"_scrapedAt":"2026-03-28T01:28:21.314Z","_scrapedBy":"cloudflare-swarm","_wikipedia":null,"indications":{"approved":[{"name":"Maroteaux-Lamy syndrome (mucopolysaccharidosis VI)"}]},"trialDetails":[{"nctId":"NCT00104234","phase":"PHASE3","title":"Study of rhASB in Patients With Mucopolysaccharidosis VI","status":"COMPLETED","sponsor":"BioMarin Pharmaceutical","startDate":"2004-02","conditions":"Mucopolysaccharidosis VI","enrollment":39},{"nctId":"NCT00067470","phase":"PHASE3","title":"Study of Recombinant Human N-acetylgalactosamine 4-sulfatase (rhASB) in Patients With MPS VI","status":"COMPLETED","sponsor":"BioMarin Pharmaceutical","startDate":"2003-09","conditions":"Mucopolysaccharidosis VI","enrollment":39}],"_emaApprovals":[],"_faersSignals":[],"_approvalHistory":[],"publicationCount":1,"rwe":[],"genericFilers":[],"relatedDrugs":[],"labelChanges":[],"biosimilarFilings":[],"pricing":[],"formularyStatus":[],"manufacturing":[],"companionDiagnostics":[],"competitors":[],"timeline":[],"patents":[],"ownershipHistory":[],"trials":[],"biosimilars":[],"latestUpdates":[],"references":[],"tags":[],"ecosystem":[],"genericManufacturerList":[],"offLabel":[],"developmentCodes":[],"aliases":[],"phase":"phase_3","status":"active","brandName":"Placebo/rhASB","genericName":"Placebo/rhASB","companyName":"BioMarin Pharmaceutical","companyId":"biomarin-pharmaceutical","modality":"Small molecule","firstApprovalDate":"","aiSummary":"rhASB (recombinant human arylsulfatase B) is an enzyme replacement therapy that replaces deficient arylsulfatase B to break down glycosaminoglycans accumulated in Maroteaux-Lamy syndrome. Used for Maroteaux-Lamy syndrome (mucopolysaccharidosis VI).","enrichmentLevel":3,"visitCount":1,"trialStats":{"total":0,"withResults":0},"verificationStatus":"verified","dataCompleteness":{"mechanism":true,"indications":true,"safety":true,"trials":true,"score":4}}