An Intravenous Brain-Penetrant Enzyme Therapy for Mucopolysaccharidosis II.

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Bibliographic Details
Title: An Intravenous Brain-Penetrant Enzyme Therapy for Mucopolysaccharidosis II.
Authors: Muenzer J; University of North Carolina School of Medicine, Chapel Hill., Burton BK; Ann and Robert H. Lurie Children's Hospital of Chicago, Northwestern University, Chicago., Harmatz P; UCSF Benioff Children's Hospital, Oakland, CA., Rajan DS; UPMC Children's Hospital of Pittsburgh, University of Pittsburgh, Pittsburgh., Jones SA; Manchester University NHS Foundation Trust, Manchester, United Kingdom., van den Hout JMP; Erasmus University Medical Center, Rotterdam, the Netherlands., Mitchell JJ; Montreal Children's Hospital, McGill University Health Centre, Montreal., Bhalla A; Denali Therapeutics, South San Francisco, CA., Engmann NJ; Denali Therapeutics, South San Francisco, CA., Zubizarreta I; Denali Therapeutics, South San Francisco, CA., Dong W; Denali Therapeutics, South San Francisco, CA., Model F; Denali Therapeutics, South San Francisco, CA., Watts RJ; Denali Therapeutics, South San Francisco, CA., Troyer MD; Denali Therapeutics, South San Francisco, CA., Chin PS; Denali Therapeutics, South San Francisco, CA., Ho C; Denali Therapeutics, South San Francisco, CA.
Source: The New England journal of medicine [N Engl J Med] 2026 Jan 01; Vol. 394 (1), pp. 39-50.
Publication Type: Journal Article; Clinical Trial, Phase II; Clinical Trial, Phase I; Multicenter Study
Journal Info: Publisher: Massachusetts Medical Society Country of Publication: United States NLM ID: 0255562 Publication Model: Print Cited Medium: Internet ISSN: 1533-4406 (Electronic) Linking ISSN: 00284793 NLM ISO Abbreviation: N Engl J Med Subsets: MEDLINE
Database: MEDLINE Ultimate
Description
ISSN:1533-4406
DOI:10.1056/NEJMoa2508681