Suchergebnisse - "Shieh, Perry B"

PubPharm (21)

1

Safety and efficacy of losmapimod in facioscapulohumeral muscular dystrophy (ReDUX4) : a randomised, double-blind, placebo-controlled phase 2b trial

enthalten in: The Lancet. Neurology | 2024

von Tawil, R. | Wagner, K. | Hamel, J. | +36

2

Efficacy and Safety of Vamorolone Over 48 Weeks in Boys With Duchenne Muscular Dystrophy : A Randomized Controlled Trial

enthalten in: Neurology | 2024

von Dang, U. | Damsker, J. | Guglieri, M. | +41

3

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

4

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

5

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

6

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

7

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

8

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

9

Safety and efficacy of avalglucosidase alfa versus alglucosidase alfa in patients with late-onset Pompe disease (COMET) : a phase 3, randomised, multicentre trial

enthalten in: The Lancet. Neurology | 2021

von Diaz-Manera, J. | Kishnani, P. | Kushlaf, H. | +127

CommentIn: Lancet Neurol. 2021 Dec;20(12):973-975. - PMID 34800404

10

Long-term efficacy and safety of dichlorphenamide for treatment of primary periodic paralysis

enthalten in: Muscle & nerve | 2021

von Sansone, V. | Johnson, N. | Hanna, M. | +5