학술논문
Repeated intravenous cardiosphere-derived cell therapy in late-stage Duchenne muscular dystrophy (HOPE-2): a multicentre, randomised, double-blind, placebo-controlled, phase 2 trial
Document Type
Article
Author
McDonald, Craig M; Marbán, Eduardo; Hendrix, Suzanne; Hogan, Nathaniel; Ruckdeschel Smith, Rachel; Eagle, Michelle; Finkel, Richard S; Tian, Cuixia; Janas, Joanne; Harmelink, Matthew M; Varadhachary, Arun S; Taylor, Michael D; Hor, Kan N; Mayer, Oscar H; Henricson, Erik K; Furlong, Pat; Ascheim, Deborah D; Rogy, Siegfried; Williams, Paula; Marbán, Linda; Butterfield, Russell; Connolly, Anne; Muntoni, Francesco; Joyce, Nanette C.; Evans, Maya; Abedi, Mehrdad; Surampudi, Prasanth; Jhawar, Sanjay; Dayan, Jonathan G.; Anthonisen, Colleen; Goude, Erica; Nicorici, Alina; Sarwary, Omaid; Prasad, Poonam; Baek, Jayoon; Newton, Andrew; Johnson, Hannah; Kusmik, Kyle; Filar, Lauri; Edmondson, Angie; Rybalsky, Irina; Chouteau, Wendy; Giordano, Anthony F.; Rodriguez, Aixa; Anderson, Kristan; Wezel, Germaine; Vega, Melisa; Duke, Julie; Collado, Jorge; Civitello, Matthew; Wells, Julie; Pyzik, Erika; Rehborg, Rebecca; Brown, Michelle; Van Eyk, Jennifer; Rogers, Russell G.
Source
The Lancet; March 2022, Vol. 399 Issue: 10329 p1049-1058, 10p
Subject
Language
ISSN
01406736; 1474547X
Abstract
Cardiosphere-derived cells (CDCs) ameliorate skeletal and cardiac muscle deterioration in experimental models of Duchenne muscular dystrophy. The HOPE-2 trial examined the safety and efficacy of sequential intravenous infusions of human allogeneic CDCs in late-stage Duchenne muscular dystrophy.