32: Idursulfase beta improves mobility and reduces organomegaly in MPS II

Idursulfase Beta — A New Therapeutic Option for MPS II with Strong Clinical Evidence Article title:Efficacy and safety of idursulfase beta in the treatment of mucopolysaccharidosis II: a phase 3, two-part study compared to a historical placebo cohort Journal:Genetics in Medicine DOI:10.1016/j.gim.2025.101460 Reference:Sohn YB, Yang A, Kim MS, Kim J, Kim JS, Oh Y, Jin DK. Efficacy and safety of idursulfase beta in the treatment of mucopolysaccharidosis II: a phase 3, two-part study compared to a historical placebo cohort. Genetics in Medicine. 2025:101460. doi:10.1016/j.gim.2025.101460 Music:Enjoy the music based on this article at the end of the episode. License:This episode is based on an open-access article published under the Creative Commons Attribution 4.0 International License (CC BY 4.0) – https://creativecommons.org/licenses/by/4.0/ Support:Base by Base – Stripe donations: https://donate.stripe.com/7sY4gz71B2sN3RWac5gEg00 Official website https://basebybase.com On PaperCast Base by Base you’ll discover the latest in genomics, functional genomics, structural genomics, and proteomics. Episode link: https://basebybase.com/episodes/idursulfase-beta-a-new-therapeutic-option-for-mps-ii-with-strong-clinical-evidence QC:This episode was checked against the original article PDF and publication metadata for the episode release published on 2025-06-05. QC Scope:- article metadata and core scientific claims from the narration- excludes analogies, intro/outro, and music- transcript coverage: Audited the phase 3 two-part trial design, primary/secondary endpoints (6-MWT, GAGs/HS/DS, liver/spleen volumes, FVC), safety/ADAs/NAbs, PK, and manufacturing differences as depicted in the transcript.- transcript topics: Phase 3 two-part trial design with historical placebo; 6-minute walk test efficacy; Urine GAG/HS/DS reductions; Liver and spleen volume changes; Pulmonary function (absolute FVC); Immunogenicity: anti-drug antibodies and neutralizing antibodies QC Summary:- factual score: 10/10- metadata score: 10/10- supported core claims: 8- claims flagged for review: 0- metadata checks passed: 4- metadata issues found: 0 Metadata Audited:- article_doi- article_title- article_journal- license Factual Items Audited:- 6-MWT improvement of 62.2 meters with idursulfase beta vs 7.3 meters with historical placebo- Urine total GAG reduction of -71.13% with idursulfase beta vs +21.39% with historical placebo- Liver volume reduction of -26.67% with idursulfase beta vs -0.80% with historical placebo- Spleen volume reduction of -26.46% with idursulfase beta vs +7.2% with historical placebo- Absolute FVC change showed no significant difference (p = 0.79)- Anti-drug antibodies detected in most participants; persistent NAb ~17% vs ~62.5% in comparator QC result: Pass.