Suchergebnisse - "Mah, Jean K"

PubPharm (25)

1

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

2

Bi-allelic variants in neuronal cell adhesion molecule cause a neurodevelopmental disorder characterized by developmental delay, hypotonia, neuropathy/spasticity

enthalten in: American journal of human genetics | 2022

von Kurolap, A. | Kreuder, F. | Gonzaga-Jauregui, C. | +37

3

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

4

Health related quality of life in young, steroid-naïve boys with Duchenne muscular dystrophy

enthalten in: Neuromuscular disorders : NMD | 2021

von Campbell, C. | McColl, E. | McDermott, M. | +45

5

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

6

Corneal Confocal Microscopy Predicts the Development of Diabetic Neuropathy : A Longitudinal Diagnostic Multinational Consortium Study

enthalten in: Diabetes care | 2021

von Perkins, B. | Lovblom, L. | Lewis, E. | +19

7

CHRNB1-associated congenital myasthenia syndrome : Expanding the clinical spectrum

enthalten in: American journal of medical genetics. Part A | 2021

von Freed, A. | Schwarz, A. | Brei, B. | +7

8

The CINRG Becker Natural History Study : Baseline characteristics

enthalten in: Muscle & nerve | 2020

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

9

Suitability of external controls for drug evaluation in Duchenne muscular dystrophy

enthalten in: Neurology | 2020

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

10

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