Interim analysis of a phase 1/2 study of weekly intravenous DNL310 (brain-penetrant enzyme replacement therapy) in mucopolysaccharidosis type II
- Resource Type
- Abstract
- Authors
- Muenzer, Joseph; Burton, Barbara K.; Harmatz, Paul; Rajan, Deepa; Jones, Simon A.; van den Hout, Hannerieke (.J).P.; Mitchell, John; Meirelles, Katia; Engmann, Natalie; Scheller, Adam; Caprihan, Rupa; Bhalla, Akhil; Hung, Tony; Nachtigall, Jason; Zubizarreta, Imanol; Lu, Sam; Zhu, Yuda; Chin, Peter; Troyer, Matt D.; Ho, Carole; Dong, Wei; Model, Fabian; Bakardjiev, Anna I.
- Source
- In Molecular Genetics and Metabolism February 2024 141(2)
- Subject
- Language
- ISSN
- 1096-7192