1. Ataluren in patients with nonsense mutation Duchenne muscular dystrophy (ACT DMD): a multicentre, randomised, double-blind, placebo-controlled, phase 3 trial. Issue 10101 (23rd September 2017) Authors: McDonald, Craig M; Campbell, Craig; Torricelli, Ricardo Erazo; Finkel, Richard S; Flanigan, Kevin M; Goemans, Nathalie; Heydemann, Peter; Kaminska, Anna; Kirschner, Janbernd; Muntoni, Francesco; Osorio, Andrés Nascimento; Schara, Ulrike; Sejersen, Thomas; Shieh, Perry B; Sweeney, H Lee; Topaloglu... Journal: Lancet Issue: Volume 390:Issue 10101(2017) Page Start: 1489 Record Type: Journal Article View Content: Available online (eLD content is only available in our Reading Rooms) ↗
2. Ataluren Pharmacokinetics in Healthy Japanese and Caucasian Subjects. Issue 2 (10th January 2019) Authors: Kong, Ronald; Laskin, Oscar L.; Kaushik, Diksha; Jin, Fengbin; Ma, Jiyuan; McIntosh, Joseph; Souza, Marcio; Almstead, Neil Journal: Clinical pharmacology in drug development Issue: Volume 8:Issue 2(2019) Page Start: 172 Record Type: Journal Article View Content: Available online (eLD content is only available in our Reading Rooms) ↗
3. Deflazacort versus prednisone/prednisolone for maintaining motor function and delaying loss of ambulation: A post HOC analysis from the ACT DMD trial. Issue 5 (27th September 2018) Authors: Shieh, Perry B.; M intosh, Joseph; Jin, Fengbin; Souza, Marcio; Elfring, Gary; Narayanan, Siva; Trifillis, Panayiota; Peltz, Stuart W.; M donald, Craig M.; Darras, Basil T. Journal: Muscle & nerve Issue: Volume 58:Issue 5(2018) Page Start: 639 Record Type: Journal Article View Content: Available online (eLD content is only available in our Reading Rooms) ↗
4. Deflazacort vs prednisone treatment for Duchenne muscular dystrophy: A meta‐analysis of disease progression rates in recent multicenter clinical trials. Issue 1 (7th November 2019) Authors: 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 Journal: Muscle & nerve Issue: Volume 61:Issue 1(2020) Page Start: 26 Record Type: Journal Article View Content: Available online (eLD content is only available in our Reading Rooms) ↗