Concepedia

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, Richard S. Finkel, Kevin M. Flanigan, Nathalie Goemans, Peter Heydemann, Anna Kamińska, Janbernd Kirschner, Francesco Muntoni,

The Lancet · 2017 · 455 citations · 23 references

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23

No Adjustments Are Needed for Multiple Comparisons

Kenneth J. Rothman · Epidemiology · 1990 · 5.8K citations

, , +15

Diagnosis and management of Duchenne muscular dystrophy, part 1: diagnosis, and pharmacological and psychosocial management

K. Bushby, Richard S. Finkel, David J. Birnkrant et al. · The Lancet Neurology · 2009 · 2K citations

, , +13

PTC124 targets genetic disorders caused by nonsense mutations

Ellen Welch, Elisabeth R. Barton, Jin Zhuo et al. · Nature · 2007 · 1.1K citations

, , +8

Duchenne Muscular Dystrophy

Craig M. McDonald, Richard T. Abresch, Gregory T. Carter et al. · American Journal of Physical Medicine & Rehabilitation · 1995 · 1.1K citations

, , +13

Survival in Duchenne muscular dystrophy: improvements in life expectancy since 1967 and the impact of home nocturnal ventilation

Michelle Eagle, Simon Baudouin, Colin Chandler et al. · Neuromuscular Disorders · 2002 · 1K citations

, , +5