Real-world and natural history data for drug evaluation in Duchenne muscular dystrophy: suitability of the North Star Ambulatory Assessment for comparisons with external controls

Author:

Muntoni Francesco,Signorovitch James,Sajeev Gautam,Goemans NathalieORCID,Wong BrendaORCID,Tian Cuixia,Mercuri Eugenio,Done NicolaeORCID,Wong Hallee,Moss Jackson,Yao Zhiwen,Ward Susan J.,Manzur Adnan,Servais Laurent,Niks Erik H.ORCID,Straub Volker,de Groot Imelda JM,McDonald Craig

Publisher

Elsevier BV

Subject

Genetics (clinical),Neurology (clinical),Neurology,Pediatrics, Perinatology and Child Health

Reference51 articles.

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5. Viewpoint: randomised controlled trials using invasive ‘placebo’ controls are unethical and should be excluded from Cochrane Reviews;Cyna;Cochrane Database Syst Rev,2011

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