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

Longitudinal ophthalmic findings in a child with Helsmoortel-Van der Aa Syndrome

por Gale, Michael J., Titus, Hope E., Harman, Gareth A., Alabduljalil, Talal, Dennis, Anna, Wilson, Jenny L., Koeller, David M., Finanger, Erika, Blasco, Peter A., Chiang, Pei-Wen, Karr, Daniel J., Yang, Paul
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

Duvoglustat HCl Increases Systemic and Tissue Exposure of Active Acid α-Glucosidase in Pompe Patients Co-administered with Alglucosidase α

por Kishnani, Priya, Tarnopolsky, Mark, Roberts, Mark, Sivakumar, Kumarswamy, Dasouki, Majed, Dimachkie, Mazen M., Finanger, Erika, Goker-Alpan, Ozlem, Guter, Karl A., Mozaffar, Tahseen, Pervaiz, Muhammad Ali, Laforet, Pascal, Levine, Todd, Adera, Matthews, Lazauskas, Richard, Sitaraman, Sheela, Khanna, Richie, Benjamin, Elfrida, Feng, Jessie, Flanagan, John J., Barth, Jay, Barlow, Carrolee, Lockhart, David J., Valenzano, Kenneth J., Boudes, Pol, Johnson, Franklin K., Byrne, Barry
Publicado 2017

Recruitment & retention program for the NeuroNEXT SMA Biomarker Study: Super Babies for SMA!

por Bartlett, Amy, Kolb, Stephen J., Kingsley, Allison, Swoboda, Kathryn J., Reyna, Sandra P., Sakonju, Ai, Darras, Basil T., Shell, Richard, Kuntz, Nancy, Castro, Diana, Iannaccone, Susan T., Parsons, Julie, Connolly, Anne M., Chiriboga, Claudia A., McDonald, Craig, Burnette, W. Bryan, Werner, Klaus, Thangarajh, Mathula, Shieh, Perry B., Finanger, Erika, Coffey, Christopher S., Yankey, Jon W., Cudkowicz, Merit E., McGovern, Michelle M., McNeil, D. Elizabeth, Arnold, W. David, Kissel, John T.
Publicado 2018

A Randomized, Double-Blind, Placebo-Controlled, Global Phase 3 Study of Edasalonexent in Pediatric Patients with Duchenne Muscular Dystrophy: Results of the PolarisDMD Trial

por Finkel, Richard S., McDonald, Craig M., Lee Sweeney, H., Finanger, Erika, Neil Knierbein, Erin, Wagner, Kathryn R., Mathews, Katherine D., Marks, Warren, Statland, Jeffrey, Nance, Jessica, McMillan, Hugh J., McCullagh, Gary, Tian, Cuixia, Ryan, Monique M., O’Rourke, Declan, Müller-Felber, Wolfgang, Tulinius, Mar, Burnette, W. Bryan, Nguyen, Cam-Tu, Vijayakumar, Kayal, Johannsen, Jessika, Phan, Han C., Eagle, Michelle, MacDougall, James, Mancini, Maria, Donovan, Joanne M.
Publicado 2021

Baseline results of the NeuroNEXT spinal muscular atrophy infant biomarker study

por Kolb, Stephen J., Coffey, Christopher S., Yankey, Jon W., Krosschell, Kristin, Arnold, W. David, Rutkove, Seward B., Swoboda, Kathryn J., Reyna, Sandra P., Sakonju, Ai, Darras, Basil T., Shell, Richard, Kuntz, Nancy, Castro, Diana, Iannaccone, Susan T., Parsons, Julie, Connolly, Anne M., Chiriboga, Claudia A., McDonald, Craig, Burnette, W. Bryan, Werner, Klaus, Thangarajh, Mathula, Shieh, Perry B., Finanger, Erika, Cudkowicz, Merit E., McGovern, Michelle M., McNeil, D. Elizabeth, Finkel, Richard, Kaye, Edward, Kingsley, Allison, Renusch, Samantha R., McGovern, Vicki L., Wang, Xueqian, Zaworski, Phillip G., Prior, Thomas W., Burghes, Arthur H.M., Bartlett, Amy, Kissel, John T.
Publicado 2016