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An Intravenous Brain-Penetrant Enzyme Therapy for Mucopolysaccharidosis II.

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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
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  Data: An Intravenous Brain-Penetrant Enzyme Therapy for Mucopolysaccharidosis II.
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  Data: <searchLink fieldCode="AU" term="%22Muenzer+J%22">Muenzer J</searchLink>; University of North Carolina School of Medicine, Chapel Hill.<br /><searchLink fieldCode="AU" term="%22Burton+BK%22">Burton BK</searchLink>; Ann and Robert H. Lurie Children's Hospital of Chicago, Northwestern University, Chicago.<br /><searchLink fieldCode="AU" term="%22Harmatz+P%22">Harmatz P</searchLink>; UCSF Benioff Children's Hospital, Oakland, CA.<br /><searchLink fieldCode="AU" term="%22Rajan+DS%22">Rajan DS</searchLink>; UPMC Children's Hospital of Pittsburgh, University of Pittsburgh, Pittsburgh.<br /><searchLink fieldCode="AU" term="%22Jones+SA%22">Jones SA</searchLink>; Manchester University NHS Foundation Trust, Manchester, United Kingdom.<br /><searchLink fieldCode="AU" term="%22van+den+Hout+JMP%22">van den Hout JMP</searchLink>; Erasmus University Medical Center, Rotterdam, the Netherlands.<br /><searchLink fieldCode="AU" term="%22Mitchell+JJ%22">Mitchell JJ</searchLink>; Montreal Children's Hospital, McGill University Health Centre, Montreal.<br /><searchLink fieldCode="AU" term="%22Bhalla+A%22">Bhalla A</searchLink>; Denali Therapeutics, South San Francisco, CA.<br /><searchLink fieldCode="AU" term="%22Engmann+NJ%22">Engmann NJ</searchLink>; Denali Therapeutics, South San Francisco, CA.<br /><searchLink fieldCode="AU" term="%22Zubizarreta+I%22">Zubizarreta I</searchLink>; Denali Therapeutics, South San Francisco, CA.<br /><searchLink fieldCode="AU" term="%22Dong+W%22">Dong W</searchLink>; Denali Therapeutics, South San Francisco, CA.<br /><searchLink fieldCode="AU" term="%22Model+F%22">Model F</searchLink>; Denali Therapeutics, South San Francisco, CA.<br /><searchLink fieldCode="AU" term="%22Watts+RJ%22">Watts RJ</searchLink>; Denali Therapeutics, South San Francisco, CA.<br /><searchLink fieldCode="AU" term="%22Troyer+MD%22">Troyer MD</searchLink>; Denali Therapeutics, South San Francisco, CA.<br /><searchLink fieldCode="AU" term="%22Chin+PS%22">Chin PS</searchLink>; Denali Therapeutics, South San Francisco, CA.<br /><searchLink fieldCode="AU" term="%22Ho+C%22">Ho C</searchLink>; Denali Therapeutics, South San Francisco, CA.
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  Data: <searchLink fieldCode="JN" term="%220255562%22">The New England journal of medicine</searchLink> [N Engl J Med] 2026 Jan 01; Vol. 394 (1), pp. 39-50.
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  Data: Journal Article; Clinical Trial, Phase II; Clinical Trial, Phase I; Multicenter Study
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  Data: <i>Publisher: </i><searchLink fieldCode="PB" term="%22Massachusetts+Medical+Society%22">Massachusetts Medical Society </searchLink><i>Country of Publication: </i>United States <i>NLM ID: </i>0255562 <i>Publication Model: </i>Print <i>Cited Medium: </i>Internet <i>ISSN: </i>1533-4406 (Electronic) <i>Linking ISSN: </i><searchLink fieldCode="IS" term="%2200284793%22">00284793 </searchLink><i>NLM ISO Abbreviation: </i>N Engl J Med <i>Subsets: </i>MEDLINE
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        Value: 10.1056/NEJMoa2508681
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        Text: English
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              Text: 2026 Jan 01
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