Suchergebnisse - "Shieh, Perry B"

PubPharm (116)

11

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

12

Efficacy and Safety of Vamorolone vs Placebo and Prednisone Among Boys With Duchenne Muscular Dystrophy : A Randomized Clinical Trial

enthalten in: JAMA neurology | 2022

von Guglieri, M. | Clemens, P. | Perlman, S. | +43

13

Quantitative Muscle Analysis in FSHD Using Whole-Body Fat-Referenced MRI : Composite Scores for Longitudinal and Cross-sectional Analysis

enthalten in: Neurology | 2022

von Mellion, M. | Widholm, P. | Karlsson, M. | +16

14

Quantitative muscle analysis in facioscapulohumeral muscular dystrophy using whole-body fat-referenced MRI : Protocol development, multicenter feasibility, and repeatability

enthalten in: Muscle & nerve | 2022

von Widholm, P. | Ahlgren, A. | Karlsson, M. | +13

15

Onasemnogene abeparvovec for presymptomatic infants with two copies of SMN2 at risk for spinal muscular atrophy type 1 : the Phase III SPR1NT trial

enthalten in: Nature medicine | 2022

von Strauss, K. | Farrar, M. | Muntoni, F. | +17

CommentIn: Nat Med. 2022 Jul;28(7):1348-1349. - PMID 35840728

16

Onasemnogene abeparvovec for presymptomatic infants with three copies of SMN2 at risk for spinal muscular atrophy : the Phase III SPR1NT trial

enthalten in: Nature medicine | 2022

von Strauss, K. | Farrar, M. | Muntoni, F. | +18

CommentIn: Nat Med. 2022 Jul;28(7):1348-1349. - PMID 35840728

17

Randomized phase 2 study of ACE-083, a muscle-promoting agent, in facioscapulohumeral muscular dystrophy

enthalten in: Muscle & nerve | 2022

von Statland, J. | Campbell, C. | Desai, U. | +26

18

Effect of Different Corticosteroid Dosing Regimens on Clinical Outcomes in Boys With Duchenne Muscular Dystrophy : A Randomized Clinical Trial

enthalten in: JAMA | 2022

von Guglieri, M. | Bushby, K. | McDermott, M. | +61

19

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

20

INCEPTUS Natural History, Run-in Study for Gene Replacement Clinical Trial in X-Linked Myotubular Myopathy

enthalten in: Journal of neuromuscular diseases | 2022

von Dowling, J. | Müller-Felber, W. | Smith, B. | +33