Phase 1 Study of Edasalonexent (CAT-1004), an Oral NF-κB Inhibitor, in Pediatric Patients with Duchenne Muscular Dystrophy

por Finanger, Erika, Vandenborne, Krista, Finkel, Richard S., Lee Sweeney, H., Tennekoon, Gihan, Yum, Sabrina, Mancini, Maria, Bista, Pradeep, Nichols, Andrew, Liu, Hanlan, Fretzen, Angelika, Donovan, Joanne M.
Publicado 2019

Intrathecal Onasemnogene Abeparvovec for Sitting, Nonambulatory Patients with Spinal Muscular Atrophy: Phase I Ascending-Dose Study (STRONG)

por Finkel, Richard S., Darras, Basil T., Mendell, Jerry R., Day, John W., Kuntz, Nancy L., Connolly, Anne M., Zaidman, Craig M., Crawford, Thomas O., Butterfield, Russell J., Shieh, Perry B., Tennekoon, Gihan, Brandsema, John F., Iannaccone, Susan T., Shoffner, John, Kavanagh, Sarah, Macek, Thomas A., Tauscher-Wisniewski, Sitra
Publicado 2023

Evaluator Training and Reliability for SMA Global Nusinersen Trials(1)

por Glanzman, Allan M., Mazzone, Elena S., Young, Sally Dunaway, Gee, Richard, Rose, Kristy, Mayhew, Anna, Nelson, Leslie, Yun, Chris, Alexander, Katie, Darras, Basil T., Zolkipli-Cunningham, Zarazuela, Tennekoon, Gihan, Day, John W., Finkel, Richard S., Mercuri, Eugenio, De Vivo, Darryl C., Baldwin, Ron, Bishop, Kathie M., Montes, Jacqueline
Publicado 2018

Magnetic Resonance Imaging and Spectroscopy Assessment of Lower Extremity Skeletal Muscles in Boys with Duchenne Muscular Dystrophy: A Multicenter Cross Sectional Study

por Forbes, Sean C., Willcocks, Rebecca J., Triplett, William T., Rooney, William D., Lott, Donovan J., Wang, Dah-Jyuu, Pollaro, Jim, Senesac, Claudia R., Daniels, Michael J., Finkel, Richard S., Russman, Barry S., Byrne, Barry J., Finanger, Erika L., Tennekoon, Gihan I., Walter, Glenn A., Sweeney, H. Lee, Vandenborne, Krista
Publicado 2014

Skeletal muscle magnetic resonance biomarkers correlate with function and sentinel events in Duchenne muscular dystrophy

por Barnard, Alison M., Willcocks, Rebecca J., Finanger, Erika L., Daniels, Michael J., Triplett, William T., Rooney, William D., Lott, Donovan J., Forbes, Sean C., Wang, Dah-Jyuu, Senesac, Claudia R., Harrington, Ann T., Finkel, Richard S., Russman, Barry S., Byrne, Barry J., Tennekoon, Gihan I., Walter, Glenn A., Sweeney, H. Lee, Vandenborne, Krista
Publicado 2018

Dominant collagen XII mutations cause a distal myopathy

por Mohassel, Payam, Liewluck, Teerin, Hu, Ying, Ezzo, Daniel, Ogata, Tracy, Saade, Dimah, Neuhaus, Sarah, Bolduc, Véronique, Zou, Yaqun, Donkervoort, Sandra, Medne, Livija, Sumner, Charlotte J., Dyck, P. James B., Wierenga, Klaas J., Tennekoon, Gihan, Finkel, Richard S., Chen, Jiani, Winder, Thomas L., Staff, Nathan P., Foley, A. Reghan, Koch, Manuel, Bönnemann, Carsten G.
Publicado 2019

Revised Hammersmith Scale for spinal muscular atrophy: A SMA specific clinical outcome assessment tool

por Ramsey, Danielle, Scoto, Mariacristina, Mayhew, Anna, Main, Marion, Mazzone, Elena S., Montes, Jacqueline, de Sanctis, Roberto, Dunaway Young, Sally, Salazar, Rachel, Glanzman, Allan M., Pasternak, Amy, Quigley, Janet, Mirek, Elizabeth, Duong, Tina, Gee, Richard, Civitello, Matthew, Tennekoon, Gihan, Pane, Marika, Pera, Maria Carmela, Bushby, Kate, Day, John, Darras, Basil T., De Vivo, Darryl, Finkel, Richard, Mercuri, Eugenio, Muntoni, Francesco
Publicado 2017

Ambulatory function in spinal muscular atrophy: Age-related patterns of progression

por Montes, Jacqueline, McDermott, Michael P., Mirek, Elizabeth, Mazzone, Elena S., Main, Marion, Glanzman, Allan M., Duong, Tina, Young, Sally Dunaway, Salazar, Rachel, Pasternak, Amy, Gee, Richard, De Sanctis, Roberto, Coratti, Giorgia, Forcina, Nicola, Fanelli, Lavinia, Ramsey, Danielle, Milev, Evelin, Civitello, Matthew, Pane, Marika, Pera, Maria Carmela, Scoto, Mariacristina, Day, John W., Tennekoon, Gihan, Finkel, Richard S., Darras, Basil T., Muntoni, Francesco, De Vivo, Darryl C., Mercuri, Eugenio
Publicado 2018