Suchergebnisse - "McDonald, Craig M"

PubPharm (9)

1

Safety and effectiveness of ataluren in patients with nonsense mutation DMD in the STRIDE Registry compared with the CINRG Duchenne Natural History Study (2015-2022) : 2022 interim analysis

enthalten in: Journal of neurology | 2023

von Mercuri, E. | Osorio, A. | Muntoni, F. | +14

ErratumIn: J Neurol. 2023 Sep;270(9):4583. - PMID 37460854

2

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

enthalten in: Journal of comparative effectiveness research | 2022

von McDonald, C. | Muntoni, F. | Penematsa, V. | +36

3

Meta-analyses of ataluren randomized controlled trials in nonsense mutation Duchenne muscular dystrophy

enthalten in: Journal of comparative effectiveness research | 2020

von Campbell, C. | Barohn, R. | Bertini, E. | +35

4

Safety and effectiveness of ataluren : comparison of results from the STRIDE Registry and CINRG DMD Natural History Study

enthalten in: Journal of comparative effectiveness research | 2020

von Mercuri, E. | Muntoni, F. | Osorio, A. | +14

CommentIn: Pediatr Neurol Briefs. 2020 Dec 04;34:12. - PMID 33304086

5

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

enthalten in: JAMA network open | 2020

von Li, D. | McDonald, C. | Elfring, G. | +4

6

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

enthalten in: Lancet (London, England) | 2017

von McDonald, C. | Campbell, C. | Torricelli, R. | +72

CommentIn: Lancet. 2017 Sep 23;390(10101):1467-1468. - PMID 28728957

7

Ataluren treatment of patients with nonsense mutation dystrophinopathy

enthalten in: Muscle & nerve | 2014

von Bushby, K. | Finkel, R. | Wong, B. | +132

8

The 6-minute walk test and other clinical endpoints in duchenne muscular dystrophy : reliability, concurrent validity, and minimal clinically important differences from a multicenter study

enthalten in: Muscle & nerve | 2013

von McDonald, C. | Henricson, E. | Abresch, R. | +56

CommentIn: Muscle Nerve. 2013 Sep;48(3):315-7. - PMID 24038058

9

The 6-minute walk test and other endpoints in Duchenne muscular dystrophy : longitudinal natural history observations over 48 weeks from a multicenter study

enthalten in: Muscle & nerve | 2013

von McDonald, C. | Henricson, E. | Abresch, R. | +56

CommentIn: Muscle Nerve. 2013 Sep;48(3):315-7. - PMID 24038058

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