An Intravenous Brain-Penetrant Enzyme Therapy for Mucopolysaccharidosis II

J Joseph Muenzer (University of North Carolina School of Medicine, Chapel Hill) B Barbara K. Burton (Ann and Robert H. Lurie Children’s Hospital of Chicago, Northwestern University, Chicago) P Paul Harmatz (UCSF Benioff Children’s Hospital Oakland, Oakland, CA) D Deepa S. Rajan (UPMC Children’s Hospital of Pittsburgh, University of Pittsburgh, Pittsburgh) S Simon A. Jones (Systems Immunity Research Institute and Division of Infection and Immunity, School of Medicine, Cardiff University) J Johanna M.P. van den Hout (Erasmus University Medical Center, Rotterdam, the Netherlands) J John J. Mitchell (Montreal Children’s Hospital, McGill University Health Centre, Montreal) A Akhil Bhalla (Denali Therapeutics, South San Francisco, CA) N Natalie J. Engmann (Denali Therapeutics, South San Francisco, CA) I Imanol Zubizarreta (Denali Therapeutics, South San Francisco, CA) W Wei Dong (Hangzhou Institute of Medicine Chinese Academy of Sciences) F Fabian Model (Denali Therapeutics, South San Francisco, CA) R Ryan J. Watts (Denali Therapeutics, South San Francisco, CA) M Matthew D. Troyer (Denali Therapeutics, South San Francisco, CA) P Peter S. Chin (Denali Therapeutics, South San Francisco, CA) C Carole Ho

Article Details

Volume / Issue Vol. 394, Issue 1
Published January 01, 2026
Pages 39-50
ISSN 0028-4793
Publisher Massachusetts Medical Society

Journal Info

New England Journal of Medicine

Massachusetts Medical Society

ISSN: 0028-4793 Social Sciences

Authors (16)

J

Joseph Muenzer

University of North Carolina School of Medicine, Chapel Hill

B

Barbara K. Burton

Ann and Robert H. Lurie Children’s Hospital of Chicago, Northwestern University, Chicago

P

Paul Harmatz

UCSF Benioff Children’s Hospital Oakland, Oakland, CA

D

Deepa S. Rajan

UPMC Children’s Hospital of Pittsburgh, University of Pittsburgh, Pittsburgh

S

Simon A. Jones

Systems Immunity Research Institute and Division of Infection and Immunity, School of Medicine, Cardiff University

J

Johanna M.P. van den Hout

Erasmus University Medical Center, Rotterdam, the Netherlands

J

John J. Mitchell

Montreal Children’s Hospital, McGill University Health Centre, Montreal

A

Akhil Bhalla

Denali Therapeutics, South San Francisco, CA

N

Natalie J. Engmann

Denali Therapeutics, South San Francisco, CA

I

Imanol Zubizarreta

Denali Therapeutics, South San Francisco, CA

W

Wei Dong

Hangzhou Institute of Medicine Chinese Academy of Sciences

F

Fabian Model

Denali Therapeutics, South San Francisco, CA

R

Ryan J. Watts

Denali Therapeutics, South San Francisco, CA

M

Matthew D. Troyer

Denali Therapeutics, South San Francisco, CA

P

Peter S. Chin

Denali Therapeutics, South San Francisco, CA

C

Carole Ho