Suchergebnisse - "Mah, Jean K"

PubPharm (11)

1

Efficacy and Safety of Vamorolone in Duchenne Muscular Dystrophy : A 30-Month Nonrandomized Controlled Open-Label Extension Trial

enthalten in: JAMA network open | 2022

von Mah, J. | Clemens, P. | Guglieri, M. | +125

2

Rasch Analysis of the Pediatric Quality of Life Inventory 4.0 Generic Core Scales Administered to Patients With Duchenne Muscular Dystrophy

enthalten in: Value in health : the journal of the International Society for Pharmacoeconomics and Outcomes Research | 2021

von Landfeldt, E. | Iff, J. | Henricson, E. | +37

3

Suitability of external controls for drug evaluation in Duchenne muscular dystrophy

enthalten in: Neurology | 2020

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

4

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

5

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

6

Disease-specific and glucocorticoid-responsive serum biomarkers for Duchenne Muscular Dystrophy

enthalten in: Scientific reports | 2019

von Hathout, Y. | Liang, C. | Ogundele, M. | +11

7

Phase IIa trial in Duchenne muscular dystrophy shows vamorolone is a first-in-class dissociative steroidal anti-inflammatory drug

enthalten in: Pharmacological research | 2018

von Conklin, L. | Damsker, J. | Hoffman, E. | +29

8

Serum pharmacodynamic biomarkers for chronic corticosteroid treatment of children

enthalten in: Scientific reports | 2016

von Hathout, Y. | Conklin, L. | Seol, H. | +12

9

Large-scale serum protein biomarker discovery in Duchenne muscular dystrophy

enthalten in: Proceedings of the National Academy of Sciences of the United States of America | 2015

von Hathout, Y. | Brody, E. | Clemens, P. | +20

10

The cooperative international neuromuscular research group Duchenne natural history study : glucocorticoid treatment preserves clinically meaningful functional milestones and reduces rate of disease progression as measured by manual muscle testing and other commonly used clinical trial outcome measures

enthalten in: Muscle & nerve | 2013

von Henricson, E. | Abresch, R. | Cnaan, A. | +110