Repeated intravenous cardiosphere-derived cell therapy in late-stage Duchenne muscular dystrophy (HOPE-2): a multicentre, randomised, double-blind, placebo-controlled, phase 2 trial

Authors: 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., 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

Source: In The Lancet 12-18 March 2022 399(10329):1049-1058

Outcomes of early‐treated infants with spinal muscular atrophy: A multicenter, retrospective cohort study.

Authors: Goedeker, Natalie L., Rogers, Amanda, Fisher, Mark, Arya, Kapil, Brandsema, John F., Farah, Hiba, Farrar, Michelle A., Felker, Marcia V., Gibbons, Melissa, Hamid, Omer Abdul, Harmelink, Matthew, Herbert, Karen, Kichula, Elizabeth, King, Kiana, Lakhotia, Arpita, Lee, Bo Hoon, Kuntz, Nancy L., Parsons, Julie, Rehborg, Rebecca, Veerapaniyan, Aravindhan

Source: Muscle & Nerve; Dec2024, Vol. 70 Issue 6, p1247-1256, 10p

Assessment of cerebral spinal fluid biomarkers and microRNA-mediated disease mechanisms in spinal muscular atrophy patient samples.

Authors: Welby, Emily, Rehborg, Rebecca J, Harmelink, Matthew, Ebert, Allison D

Source: Human Molecular Genetics; 6/1/2022, Vol. 31 Issue 11, p1830-1843, 14p

Repeated intravenous cardiosphere-derived cell therapy in late-stage Duchenne muscular dystrophy (HOPE-2): a multicentre, randomised, double-blind, placebo-controlled, phase 2 trial

Authors: 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

Validity of Respiratory Scores in Bronchiolitis.

Authors: Destino, Lauren, Weisgerber, Michael C., Soung, Paula, Bakalarski, Deborah, Ke Yan, Rehborg, Rebecca, Wagner, Duke R., Gorelick, Marc H., Simpson, Pippa

Source: Hospital Pediatrics; 10/1/2012, Vol. 2 Issue 4, p202-209, 8p