Suchergebnisse - "Shieh, Perry B"

PubPharm (29)

1

Safety and efficacy of gene replacement therapy for X-linked myotubular myopathy (ASPIRO) : a multinational, open-label, dose-escalation trial

enthalten in: The Lancet. Neurology | 2023

von Shieh, P. | Kuntz, N. | Dowling, J. | +30

CommentIn: Lancet Neurol. 2023 Dec;22(12):1089-1091. - PMID 37977700

2

Efficacy and Safety of Avalglucosidase Alfa in Patients With Late-Onset Pompe Disease After 97 Weeks : A Phase 3 Randomized Clinical Trial

enthalten in: JAMA neurology | 2023

von Kishnani, P. | Diaz-Manera, J. | Toscano, A. | +125

3

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

4

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

5

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

6

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

7

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

8

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

9

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

10

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