Suchergebnisse - "Shieh, Perry B"

PubPharm (72)

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

Effects of gene replacement therapy with resamirigene bilparvovec (AT132) on skeletal muscle pathology in X-linked myotubular myopathy : results from a substudy of the ASPIRO open-label clinical trial

enthalten in: EBioMedicine | 2024

von Lawlor, M. | Schoser, B. | Margeta, M. | +36

4

Recurrent de novo SPTLC2 variant causes childhood-onset amyotrophic lateral sclerosis (ALS) by excess sphingolipid synthesis

enthalten in: Journal of neurology, neurosurgery, and psychiatry | 2024

von Syeda, S. | Lone, M. | Mohassel, P. | +23

5

Real-World Outcomes in Patients with Spinal Muscular Atrophy Treated with Onasemnogene Abeparvovec Monotherapy : Findings from the RESTORE Registry

enthalten in: Journal of neuromuscular diseases | 2024

von Servais, L. | Day, J. | De Vivo, D. | +16

6

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

7

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

8

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

9

Intrathecal Onasemnogene Abeparvovec for Sitting, Nonambulatory Patients with Spinal Muscular Atrophy : Phase I Ascending-Dose Study (STRONG)

enthalten in: Journal of neuromuscular diseases | 2023

von Finkel, R. | Darras, B. | Mendell, J. | +14

10

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

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