Suchergebnisse - "Shieh, Perry B"

PubPharm (17)

1

Disease progression rates in ambulatory Duchenne muscular dystrophy by steroid type, patient age and functional status

enthalten in: Journal of comparative effectiveness research | 2023

von McDonald, C. | Marden, J. | Shieh, P. | +8

2

Expression of SRP-9001 dystrophin and stabilization of motor function up to 2 years post-treatment with delandistrogene moxeparvovec gene therapy in individuals with Duchenne muscular dystrophy

enthalten in: Frontiers in cell and developmental biology | 2023

von Mendell, J. | Shieh, P. | McDonald, C. | +17

3

A Combined Prospective and Retrospective Comparison of Long-Term Functional Outcomes Suggests Delayed Loss of Ambulation and Pulmonary Decline with Long-Term Eteplirsen Treatment

enthalten in: Journal of neuromuscular diseases | 2022

von Mitelman, O. | Abdel-Hamid, H. | Byrne, B. | +15

4

Meta-analyses of deflazacort versus prednisone/prednisolone in patients with nonsense mutation Duchenne muscular dystrophy

enthalten in: Journal of comparative effectiveness research | 2021

von Shieh, P. | Elfring, G. | Trifillis, P. | +111

5

Health related quality of life in young, steroid-naïve boys with Duchenne muscular dystrophy

enthalten in: Neuromuscular disorders : NMD | 2021

von Campbell, C. | McColl, E. | McDermott, M. | +45

6

Safety, tolerability, and pharmacokinetics of casimersen in patients with Duchenne muscular dystrophy amenable to exon 45 skipping : A randomized, double-blind, placebo-controlled, dose-titration trial

enthalten in: Muscle & nerve | 2021

von Wagner, K. | Kuntz, N. | Koenig, E. | +4

7

Disease-modifying effects of edasalonexent, an NF-κB inhibitor, in young boys with Duchenne muscular dystrophy : Results of the MoveDMD phase 2 and open label extension trial

enthalten in: Neuromuscular disorders : NMD | 2021

von Finkel, R. | Finanger, E. | Vandenborne, K. | +15

8

Deflazacort vs prednisone treatment for Duchenne muscular dystrophy : A meta-analysis of disease progression rates in recent multicenter clinical trials

enthalten in: Muscle & nerve | 2020

von McDonald, C. | Sajeev, G. | Yao, Z. | +10

9

Large in-frame 5' deletions in DMD associated with mild Duchenne muscular dystrophy : Two case reports and a review of the literature

enthalten in: Neuromuscular disorders : NMD | 2019

von Gibbs, E. | Barthélémy, F. | Douine, E. | +6

10

Emerging Strategies in the Treatment of Duchenne Muscular Dystrophy

enthalten in: Neurotherapeutics : the journal of the American Society for Experimental NeuroTherapeutics | 2018

von Shieh, P.