P026: Interim analysis of the efficacy and safety of weekly intravenous tividenofusp alfa in mucopolysaccharidosis type II: A phase 1/2 study
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| Format: | Article |
| Language: | English |
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Elsevier
2025-01-01
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| Series: | Genetics in Medicine Open |
| Online Access: | http://www.sciencedirect.com/science/article/pii/S2949774425009094 |
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| _version_ | 1849713319452082176 |
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| author | Barbara Burton Joseph Muenzer Paul Harmatz Deepa Rajan Simon Jones Johanna van den Hout John Mitchell Matthew Troyer Natalie Engmann Rupa Caprihan Akhil Bhalla Imanol Zubizarreta Arlette Weichert Peter Chin Carole Ho |
| author_facet | Barbara Burton Joseph Muenzer Paul Harmatz Deepa Rajan Simon Jones Johanna van den Hout John Mitchell Matthew Troyer Natalie Engmann Rupa Caprihan Akhil Bhalla Imanol Zubizarreta Arlette Weichert Peter Chin Carole Ho |
| author_sort | Barbara Burton |
| collection | DOAJ |
| format | Article |
| id | doaj-art-b8bc47e1206942bf8d4518ffe05e011b |
| institution | DOAJ |
| issn | 2949-7744 |
| language | English |
| publishDate | 2025-01-01 |
| publisher | Elsevier |
| record_format | Article |
| series | Genetics in Medicine Open |
| spelling | doaj-art-b8bc47e1206942bf8d4518ffe05e011b2025-08-20T03:14:00ZengElsevierGenetics in Medicine Open2949-77442025-01-01310287010.1016/j.gimo.2025.102870P026: Interim analysis of the efficacy and safety of weekly intravenous tividenofusp alfa in mucopolysaccharidosis type II: A phase 1/2 studyBarbara Burton0Joseph Muenzer1Paul Harmatz2Deepa Rajan3Simon Jones4Johanna van den Hout5John Mitchell6Matthew Troyer7Natalie Engmann8Rupa Caprihan9Akhil Bhalla10Imanol Zubizarreta11Arlette Weichert12Peter Chin13Carole Ho14Ann & Robert H Lurie Children’s Hospital of Chicago, Northwestern University, Chicago, ILUniversity of North Carolina School of Medicine, Chapel Hill, NCUCSF Benioff Children’s Hospital, Oakland, CAUPMC Children’s Hospital of Pittsburgh, Pittsburgh, PAManchester University National Health Service Foundation Trust, Manchester, UKErasmus University Medical Center, Rotterdam, NetherlandsMontreal Children’s Hospital, McGill University Health Centre, Montreal, QC, CanadaDenali Therapeutics Inc, South San Francisco, CADenali Therapeutics Inc, South San Francisco, CADenali Therapeutics Inc, South San Francisco, CADenali Therapeutics Inc, South San Francisco, CADenali Therapeutics Inc, South San Francisco, CADenali Therapeutics Inc, South San Francisco, CADenali Therapeutics Inc, South San Francisco, CADenali Therapeutics Inc, South San Francisco, CAhttp://www.sciencedirect.com/science/article/pii/S2949774425009094 |
| spellingShingle | Barbara Burton Joseph Muenzer Paul Harmatz Deepa Rajan Simon Jones Johanna van den Hout John Mitchell Matthew Troyer Natalie Engmann Rupa Caprihan Akhil Bhalla Imanol Zubizarreta Arlette Weichert Peter Chin Carole Ho P026: Interim analysis of the efficacy and safety of weekly intravenous tividenofusp alfa in mucopolysaccharidosis type II: A phase 1/2 study Genetics in Medicine Open |
| title | P026: Interim analysis of the efficacy and safety of weekly intravenous tividenofusp alfa in mucopolysaccharidosis type II: A phase 1/2 study |
| title_full | P026: Interim analysis of the efficacy and safety of weekly intravenous tividenofusp alfa in mucopolysaccharidosis type II: A phase 1/2 study |
| title_fullStr | P026: Interim analysis of the efficacy and safety of weekly intravenous tividenofusp alfa in mucopolysaccharidosis type II: A phase 1/2 study |
| title_full_unstemmed | P026: Interim analysis of the efficacy and safety of weekly intravenous tividenofusp alfa in mucopolysaccharidosis type II: A phase 1/2 study |
| title_short | P026: Interim analysis of the efficacy and safety of weekly intravenous tividenofusp alfa in mucopolysaccharidosis type II: A phase 1/2 study |
| title_sort | p026 interim analysis of the efficacy and safety of weekly intravenous tividenofusp alfa in mucopolysaccharidosis type ii a phase 1 2 study |
| url | http://www.sciencedirect.com/science/article/pii/S2949774425009094 |
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