Literature DB >> 32058550

Assessment of Treatment Effect With Multiple Outcomes in 2 Clinical Trials of Patients With Duchenne Muscular Dystrophy.

Daniel Li1, Craig M McDonald2, Gary L Elfring3, Marcio Souza3, Joseph McIntosh3, Dae Hyun Kim4, Lee-Jen Wei1.   

Abstract

Entities:  

Year:  2020        PMID： 32058550      PMCID： PMC7202370          DOI： 10.1001/jamanetworkopen.2019.21306

Source DB:  PubMed          Journal:  JAMA Netw Open        ISSN： 2574-3805


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  3 in total

1.  Methods for the analysis of multiple endpoints in small populations: A review.

Authors:  Robin Ristl; Susanne Urach; Gerd Rosenkranz; Martin Posch
Journal:  J Biopharm Stat       Date:  2018-07-09       Impact factor: 1.051

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

Authors:  Craig M McDonald; Craig Campbell; Ricardo Erazo Torricelli; Richard S Finkel; Kevin M Flanigan; Nathalie Goemans; Peter Heydemann; Anna Kaminska; Janbernd Kirschner; Francesco Muntoni; Andrés Nascimento Osorio; Ulrike Schara; Thomas Sejersen; Perry B Shieh; H Lee Sweeney; Haluk Topaloglu; Már Tulinius; Juan J Vilchez; Thomas Voit; Brenda Wong; Gary Elfring; Hans Kroger; Xiaohui Luo; Joseph McIntosh; Tuyen Ong; Peter Riebling; Marcio Souza; Robert J Spiegel; Stuart W Peltz; Eugenio Mercuri
Journal:  Lancet       Date:  2017-07-17       Impact factor: 79.321

3.  Ataluren treatment of patients with nonsense mutation dystrophinopathy.

Authors:  Katharine Bushby; Richard Finkel; Brenda Wong; Richard Barohn; Craig Campbell; Giacomo P Comi; Anne M Connolly; John W Day; Kevin M Flanigan; Nathalie Goemans; Kristi J Jones; Eugenio Mercuri; Ros Quinlivan; James B Renfroe; Barry Russman; Monique M Ryan; Mar Tulinius; Thomas Voit; Steven A Moore; H Lee Sweeney; Richard T Abresch; Kim L Coleman; Michelle Eagle; Julaine Florence; Eduard Gappmaier; Allan M Glanzman; Erik Henricson; Jay Barth; Gary L Elfring; Allen Reha; Robert J Spiegel; Michael W O'donnell; Stuart W Peltz; Craig M Mcdonald
Journal:  Muscle Nerve       Date:  2014-10       Impact factor: 3.217
  3 in total
  4 in total

Review 1.  Review of neuropsychological outcomes in isolated methylmalonic acidemia: recommendations for assessing impact of treatments.

Authors:  Susan E Waisbren
Journal:  Metab Brain Dis       Date:  2022-03-29       Impact factor: 3.655

2.  Read-through approach for stop mutations in Duchenne muscular dystrophy. An update.

Authors:  Luisa Politano
Journal:  Acta Myol       Date:  2021-03-31

3.  Ataluren delays loss of ambulation and respiratory decline in nonsense mutation Duchenne muscular dystrophy patients.

Authors:  Craig M McDonald; Francesco Muntoni; Vinay Penematsa; Joel Jiang; Allan Kristensen; Francesco Bibbiani; Elizabeth Goodwin; Heather Gordish-Dressman; Lauren Morgenroth; Christian Werner; James Li; Richard Able; Panayiota Trifillis; Már Tulinius
Journal:  J Comp Eff Res       Date:  2021-11-18       Impact factor: 2.040

Review 4.  [Expert recommendation: treatment of nonambulatory patients with Duchenne muscular dystrophy].

Authors:  Guenther Bernert; Andreas Hahn; Cornelia Köhler; Sascha Meyer; Ulrike Schara; Kurt Schlachter; Regina Trollmann; Maggie C Walter
Journal:  Nervenarzt       Date:  2020-11-19       Impact factor: 1.214
  4 in total

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