Results from a phase 1 study of nusinersen (ISIS-SMNRx) in children with spinal muscular atrophy

by Chiriboga, Claudia A., Swoboda, Kathryn J., Darras, Basil T., Iannaccone, Susan T., Montes, Jacqueline, De Vivo, Darryl C., Norris, Daniel A., Bennett, C. Frank, Bishop, Kathie M.
Published 2016

SMA-MAP: A Plasma Protein Panel for Spinal Muscular Atrophy

by Kobayashi, Dione T., Shi, Jing, Stephen, Laurie, Ballard, Karri L., Dewey, Ruth, Mapes, James, Chung, Brett, McCarthy, Kathleen, Swoboda, Kathryn J., Crawford, Thomas O., Li, Rebecca, Plasterer, Thomas, Joyce, Cynthia, Chung, Wendy K., Kaufmann, Petra, Darras, Basil T., Finkel, Richard S., Sproule, Douglas M., Martens, William B., McDermott, Michael P., De Vivo, Darryl C., Walker, Michael G., Chen, Karen S.
Published 2013

Developmental milestones in type I spinal muscular atrophy

by De Sanctis, Roberto, Coratti, Giorgia, Pasternak, Amy, Montes, Jacqueline, Pane, Marika, Mazzone, Elena S., Young, Sally Dunaway, Salazar, Rachel, Quigley, Janet, Pera, Maria C., Antonaci, Laura, Lapenta, Leonardo, Glanzman, Allan M., Tiziano, Danilo, Muntoni, Francesco, Darras, Basil T., De Vivo, Darryl C., Finkel, Richard, Mercuri, Eugenio
Published 2016

Patterns of disease progression in type 2 and 3 SMA: Implications for clinical trials

by Mercuri, Eugenio, Finkel, Richard, Montes, Jacqueline, Mazzone, Elena S., Sormani, Maria Pia, Main, Marion, Ramsey, Danielle, Mayhew, Anna, Glanzman, Allan M., Dunaway, Sally, Salazar, Rachel, Pasternak, Amy, Quigley, Janet, Pane, Marika, Pera, Maria Carmela, Scoto, Mariacristina, Messina, Sonia, Sframeli, Maria, Vita, Gian Luca, D'Amico, Adele, van den Hauwe, Marleen, Sivo, Serena, Goemans, Nathalie, Kaufmann, Petra, Darras, Basil T., Bertini, Enrico, Muntoni, Francesco, De Vivo, Darryl C.
Published 2016