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

द्वारा 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
प्रकाशित 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...

द्वारा 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
प्रकाशित 2013

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

द्वारा 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.
में प्रकाशित Clin Pharmacol Drug Dev (2016)

A randomized placebo-controlled trial of ataluren for the treatment of nonsense mutation cystic fibrosis

द्वारा EitanKerem, Konstan, Michael W., De Boeck, Kris, Accurso, Frank J., Sermet-Gaudelus, Isabelle, Wilschanski, Michael, Elborn, J S, Melotti, Paola, Bronsveld, Inez, Fajac, Isabelle, Malfroot, Anne, Rosenbluth, Daniel B., Walker, Patricia A., McColley, Susanna A., Knoop, Christiane, Quattrucci, Serena, Rietschel, Ernst, Zeitlin, Pamela L., Barth, Jay, Elfring, Gary L., Welch, Ellen M., Branstrom, Arthur, Spiegel, Robert J., Peltz, Stuart W., Ajayi, Temitayo, Rowe, Steven M.
प्रकाशित 2014

Ataluren treatment of patients with nonsense mutation dystrophinopathy

द्वारा 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
में प्रकाशित Muscle Nerve (2014)