Publications

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Delivery of acid a-glucosidase by muscle-targeting extracellular vesicles: a new road for Pompe disease treatment

Li-En Hsieh, Mafalda Cacciottolo, Daniel Leychkis, Wenny Wong, Bailey Murphy, Huilan Lin, Christy Liu, Nicole Bee, Linda Marban, Kristi Elliott, Minghao Sun

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Engineered muscle-targeting extracellular vesicles for systemic delivery of micro-dystrophin: novel redosable strategy for Duchenne muscular dystrophy.

David Tran, Meena Murali, Christy Lau, Bailey Murphy, Huilan Lin, Firouz Mohsenian, Yujia Li, Mafalda Cacciottolo, Linda Marban, Kristi Elliott, Minghao Sun

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HOPE-3, a phase 3 study of deramiocel, an allogeneic cell therapy, in advanced Duchenne muscular dystrophy: evidence to support both musculoskeletal and cardiac efficacy

Craig McDonald, M.D., Chet Villa, M.D., Jon Soslow, M.D., Erik Henricson, Ph.D., Cuixia Tian, M.D., Russell Butterfield, M.D., Ph.D., Kati Maharry, Ph.D., Nathan Hogan, Ph.D., Michael Binks, M.D, Kevin Berth, Mark Awadalla, Eugenio Mercuri, M.D, Eduardo Marban, M.D., Ph.D., Linda Marban, Ph.D., and the HOPE-3 Investigators

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Deramiocel slows upper limb decline in the HOPE-3 open-label extension: cross-phase analysis and 2-year comparison with natural history

Craig M McDonald, Cuixia Tian, Aravindhan Veerapandiyan, Susan Apkon, Partha S Ghosh, Katheryn Gambetta, Saunder M Bernes, Arun S Varadhachary, Susan T Iannaccone, Chamindra G Laverty, Russell J Butterfield, Katherine D Mathews, Seth J Perlman, Nancy E Bass, Kati Maharry, Adam Sullivan, Nathaniel Hogan, Michael Binks, Mark SAwadalla, Linda Marban, and the HOPE-3 Investigators

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The Lancet Publishes HOPE-3 Data for Capricor’s Deramiocel in DMD

Craig M McDonald, Chet Villa, Jonathan H Soslow, Kati Maharry, Nathaniel Hogan, Michael Binks, Kevin C Berth, Kristi A Elliott, Michael Taylor, Kan N Hor, James Signorovich, Erik K Henricson, Han C Phan, Susan Apkon, Partha S Ghosh, Cuixia Tian, Aravindhan Veerapandiyan, Leigh Ramos-Platt, Katheryn Gambetta, Saunder M Bernes, Arun S Varadhachary, Susan T Iannaccone, Chamindra G Laverty, Seth J Perlman, Nancy E Bass, Kathryn Mosher, Russell J Butterfield, Katherine D Mathews, Rebecca J Scharf, Edward C Smith, Jane Anne Emerson, Eugenio Mercuri, Mark S Awadalla, Linda Marbán*, Eduardo Marbán*, on behalf of the HOPE-3 Investigators†

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PPMD 2026 (Late-Breaking HOPE-2 OLE 5-Year Results)

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American Society of Gene & Cell Therapy (ASGCT) 2026 Annual Meeting Presentation

Jonathan H. Soslow, Chet Villa, Russell J Butterfield, Michael Binks, Kati Maharry, Nathan Horgan, Kristi Elliott, Minghao Sun, Mark Awadalla, Linda Marban, Craig McDonald

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