Longitudinal Evaluation of Working Memory in Duchenne Muscular Dystrophy

por Thangarajh, Mathula, Elfring, Gary L., Trifillis, Panayiota
Publicado 2020

Evaluating longitudinal therapy effects via the North Star Ambulatory Assessment

por McDonald, Craig M., Wei, Lee‐Jen, Flanigan, Kevin M., Elfring, Gary, Trifillis, Panayiota, Muntoni, Francesco
Publicado 2021

Percent-Predicted 6-Minute Walk Distance in Duchenne Muscular Dystrophy to Account for Maturational Influences

por Henricson, Erik, Abresch, Richard, Han, Jay J., Nicorici, Alina, Goude Keller, Erica, Elfring, Gary, Reha, Allen, Barth, Jay, McDonald, Craig M.
Publicado 2012

Deflazacort versus prednisone/prednisolone for maintaining motor function and delaying loss of ambulation: A post HOC analysis from the ACT DMD trial

por Shieh, Perry B., Mcintosh, Joseph, Jin, Fengbin, Souza, Marcio, Elfring, Gary, Narayanan, Siva, Trifillis, Panayiota, Peltz, Stuart W., Mcdonald, Craig M., Darras, Basil T.
Publicado 2018

Phase 2a Study of Ataluren-Mediated Dystrophin Production in Patients with Nonsense Mutation Duchenne Muscular Dystrophy

por Finkel, Richard S., Flanigan, Kevin M., Wong, Brenda, Bönnemann, Carsten, Sampson, Jacinda, Sweeney, H. Lee, Reha, Allen, Northcutt, Valerie J., Elfring, Gary, Barth, Jay, Peltz, Stuart W.
Publicado 2013

THE 6-MINUTE WALK TEST AND OTHER ENDPOINTS IN DUCHENNE MUSCULAR DYSTROPHY: LONGITUDINAL NATURAL HISTORY OBSERVATIONS OVER 48 WEEKS FROM A MULTICENTER STUDY

por Mcdonald, Craig M, Henricson, Erik K, Abresch, R Ted, Florence, Julaine M, Eagle, Michelle, Gappmaier, Eduard, Glanzman, Allan M, Spiegel, Robert, Barth, Jay, Elfring, Gary, Reha, Allen, Peltz, Stuart
Publicado 2013

THE 6-MINUTE WALK TEST AND OTHER CLINICAL ENDPOINTS IN DUCHENNE MUSCULAR DYSTROPHY: RELIABILITY, CONCURRENT VALIDITY, AND MINIMAL CLINICALLY IMPORTANT DIFFERENCES FROM A MULTICENTE...

por McDonald, Craig M, Henricson, Erik K, Abresch, R Ted, Florence, Julaine, Eagle, Michelle, Gappmaier, Eduard, Glanzman, Allan M, Spiegel, Robert, Barth, Jay, Elfring, Gary, Reha, Allen, Peltz, Stuart W
Publicado 2013

Phase 1 Study of Safety, Tolerability, and Pharmacokinetics of PTC299, an Inhibitor of Stress‐Regulated Protein Translation

por Weetall, Marla, Davis, Thomas, Elfring, Gary, Northcutt, Valerie, Cao, Liangxian, Moon, Young‐Choon, Riebling, Peter, Dali, Mandar, Hirawat, Samit, Babiak, John, Colacino, Joseph, Almstead, Neil, Spiegel, Robert, Peltz, Stuart W.
Publicado 2016

Deflazacort vs prednisone treatment for Duchenne muscular dystrophy: A meta‐analysis of disease progression rates in recent multicenter clinical trials

por McDonald, Craig M., Sajeev, Gautam, Yao, Zhiwen, McDonnell, Erin, Elfring, Gary, Souza, Marcio, Peltz, Stuart W., Darras, Basil T., Shieh, Perry B., Cox, David A., Landry, John, Signorovitch, James
Publicado 2019

The minor gentamicin complex component, X2, is a potent premature stop codon readthrough molecule with therapeutic potential

por Friesen, Westley J., Johnson, Briana, Sierra, Jairo, Zhuo, Jin, Vazirani, Priya, Xue, Xiaojiao, Tomizawa, Yuki, Baiazitov, Ramil, Morrill, Christie, Ren, Hongyu, Babu, Suresh, Moon, Young-Choon, Branstrom, Art, Mollin, Anna, Hedrick, Jean, Sheedy, Josephine, Elfring, Gary, Weetall, Marla, Colacino, Joseph M., Welch, Ellen M., Peltz, Stuart W.
Publicado 2018

Ataluren treatment of patients with nonsense mutation dystrophinopathy

por Bushby, Katharine, Finkel, Richard, Wong, Brenda, Barohn, Richard, Campbell, Craig, Comi, Giacomo P, Connolly, Anne M, Day, John W, Flanigan, Kevin M, Goemans, Nathalie, Jones, Kristi J, Mercuri, Eugenio, Quinlivan, Ros, Renfroe, James B, Russman, Barry, Ryan, Monique M, Tulinius, Mar, Voit, Thomas, Moore, Steven A, Lee Sweeney, H, Abresch, Richard T, Coleman, Kim L, Eagle, Michelle, Florence, Julaine, Gappmaier, Eduard, Glanzman, Allan M, Henricson, Erik, Barth, Jay, Elfring, Gary L, Reha, Allen, Spiegel, Robert J, O'donnell, Michael W, Peltz, Stuart W, Mcdonald, Craig M, FOR THE PTC124-GD-007-DMD STUDY GROUP
Publicado 2014