Joseph McIntosh

Ataluren in patients with nonsense mutation Duchenne muscular dystrophy (ACT DMD): a multicentre, randomised, double-blind, placebo-controlled, phase 3 trial

Craig M. McDonald, Craig Campbell, Ricardo Erazo Torricelli et al. · The Lancet · 2017 · 446 citations · Full text

Rare Diseases, Urology, Clinical Trials +5

Efficacy and safety of ataluren in patients with nonsense-mutation cystic fibrosis not receiving chronic inhaled aminoglycosides: The international, randomized, double-blind, placebo-controlled Ataluren Confirmatory Trial in Cystic Fibrosis (ACT CF)

Michael W. Konstan, Donald R. VanDevanter, Steven M. Rowe et al. · Journal of Cystic Fibrosis · 2020 · 65 citations · Full text

Pulmonary Care, Act Cf, Clinical Trials +3

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

Perry B. Shieh, Joseph McIntosh, Fengbin Jin et al. · Muscle & Nerve · 2018 · 59 citations · Full text

Evaluating assumptions of definition-based pulmonary exacerbation endpoints in cystic fibrosis clinical trials

Donald R. VanDevanter, N. Hamblett, Noah Simon et al. · Journal of Cystic Fibrosis · 2020 · 18 citations · Full text

Pulmonary Care, Advanced Lung Disease, Lung Inflammation +4

The relationship between deficit in digit span and genotype in nonsense mutation Duchenne muscular dystrophy

Mathula Thangarajh, Gary Elfring, Panayiota Trifillis et al. · Neurology · 2018 · 15 citations · Full text