Suchergebnisse - "Mah, Jean K"

PubPharm (203)

41

Multi-Omics Identifies Circulating miRNA and Protein Biomarkers for Facioscapulohumeral Dystrophy

enthalten in: Journal of personalized medicine | 2020

von Heier, C. | Zhang, A. | Nguyen, N. | +19

42

A National Spinal Muscular Atrophy Registry for Real-World Evidence

enthalten in: The Canadian journal of neurological sciences. Le journal canadien des sciences neurologiques | 2020

von Hodgkinson, V. | Oskoui, M. | Lounsberry, J. | +47

43

Meta-analyses of ataluren randomized controlled trials in nonsense mutation Duchenne muscular dystrophy

enthalten in: Journal of comparative effectiveness research | 2020

von Campbell, C. | Barohn, R. | Bertini, E. | +35

44

The CINRG Becker Natural History Study : Baseline characteristics

enthalten in: Muscle & nerve | 2020

von Clemens, P. | Niizawa, G. | Feng, J. | +24

45

Suitability of external controls for drug evaluation in Duchenne muscular dystrophy

enthalten in: Neurology | 2020

von Goemans, N. | Signorovitch, J. | Sajeev, G. | +92

46

Efficacy and safety of vamorolone in Duchenne muscular dystrophy: An 18-month interim analysis of a non-randomized open-label extension study

enthalten in: PLoS medicine | 2020

von Smith, E. | Conklin, L. | Hoffman, E. | +24

47

Safety, Tolerability, and Efficacy of Viltolarsen in Boys With Duchenne Muscular Dystrophy Amenable to Exon 53 Skipping : A Phase 2 Randomized Clinical Trial

enthalten in: JAMA neurology | 2020

von Clemens, P. | Rao, V. | Connolly, A. | +11

ErratumIn: JAMA Neurol. 2020 Aug 1;77(8):1040. - PMID 32539096

48

TCTEX1D1 is a genetic modifier of disease progression in Duchenne muscular dystrophy

enthalten in: European journal of human genetics : EJHG | 2020

von Spitali, P. | Zaharieva, I. | Bohringer, S. | +59

49

Randomized phase 2 trial and open-label extension of domagrozumab in Duchenne muscular dystrophy

enthalten in: Neuromuscular disorders : NMD | 2020

von Wagner, K. | Abdel-Hamid, H. | Mah, J. | +21

ErratumIn: Neuromuscul Disord. 2021 Feb;31(2):167-168. - PMID 33451933

50

Towards regulatory endorsement of drug development tools to promote the application of model-informed drug development in Duchenne muscular dystrophy

enthalten in: Journal of pharmacokinetics and pharmacodynamics | 2019

von Conrado, D. | Larkindale, J. | Berg, A. | +120