Phase 1 Study of AAV9.LAMP2B Gene Therapy in Danon Disease

Barry Greenberg; Matthew Taylor; Eric Adler; Steven Colan; David Ricks; Paul Yarabe; Pavan Battiprolu; Gaurav Shah; Kinnari Patel; Matthew Coggins; Susanna Carou-Keenan; Jonathan D. Schwartz; Joseph W. Rossano

doi:10.1056/nejmoa2412392

Phase 1 Study of AAV9.LAMP2B Gene Therapy in Danon Disease

B Barry Greenberg (University of California, San Diego Medical Center, La Jolla) M Matthew Taylor (University of Colorado, Anschutz, Medical Center, Aurora) E Eric Adler (University of California, San Diego Medical Center, La Jolla) S Steven Colan (Boston Children’s Hospital, Harvard Medical School, Boston) D David Ricks (Rocket Pharmaceuticals, Cranbury, NJ) P Paul Yarabe (Rocket Pharmaceuticals, Cranbury, NJ) P Pavan Battiprolu (Rocket Pharmaceuticals, Cranbury, NJ) G Gaurav Shah (Rocket Pharmaceuticals, Cranbury, NJ) K Kinnari Patel (Rocket Pharmaceuticals, Cranbury, NJ) M Matthew Coggins (Rocket Pharmaceuticals, Cranbury, NJ) S Susanna Carou-Keenan (Rocket Pharmaceuticals, Cranbury, NJ) J Jonathan D. Schwartz (Rocket Pharmaceuticals, Cranbury, NJ) J Joseph W. Rossano (Division of Cardiology, Children’s Hospital of Philadelphia, Philadelphia)

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Article Details

Journal New England Journal of Medicine

Volume / Issue Vol. 392, Issue 10

Published March 06, 2025

Pages 972-983

DOI 10.1056/nejmoa2412392

ISSN 0028-4793

Publisher Massachusetts Medical Society

Journal Info

New England Journal of Medicine

Massachusetts Medical Society

ISSN: 0028-4793 Social Sciences

Authors (13)

B

Barry Greenberg

University of California, San Diego Medical Center, La Jolla

M

Matthew Taylor

University of Colorado, Anschutz, Medical Center, Aurora

E

Eric Adler

University of California, San Diego Medical Center, La Jolla

S

Steven Colan

Boston Children’s Hospital, Harvard Medical School, Boston

D

David Ricks