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1.
Efficacy and safety of vamorolone in Duchenne muscular dystrophy: An 18-month interim analysis of a non-randomized open-label extension study.
PLoS Med
; 17(9): e1003222, 2020 09.
Artículo
en Inglés
| MEDLINE | ID: mdl-32956407
2.
Phase IIa trial in Duchenne muscular dystrophy shows vamorolone is a first-in-class dissociative steroidal anti-inflammatory drug.
Pharmacol Res
; 136: 140-150, 2018 10.
Artículo
en Inglés
| MEDLINE | ID: mdl-30219580
3.
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.
Muscle Nerve
; 48(1): 55-67, 2013 Jul.
Artículo
en Inglés
| MEDLINE | ID: mdl-23649481
4.
The cooperative international neuromuscular research group Duchenne natural history study--a longitudinal investigation in the era of glucocorticoid therapy: design of protocol and the methods used.
Muscle Nerve
; 48(1): 32-54, 2013 Jul.
Artículo
en Inglés
| MEDLINE | ID: mdl-23677550
5.
Liquid formulation of pentoxifylline is a poorly tolerated treatment for duchenne dystrophy.
Muscle Nerve
; 44(2): 170-3, 2011 Aug.
Artículo
en Inglés
| MEDLINE | ID: mdl-21674534
6.
Vamorolone trial in Duchenne muscular dystrophy shows dose-related improvement of muscle function.
Neurology
; 93(13): e1312-e1323, 2019 09 24.
Artículo
en Inglés
| MEDLINE | ID: mdl-31451516
7.
Feasibility and Reproducibility of Echocardiographic Measures in Children with Muscular Dystrophies.
J Am Soc Echocardiogr
; 28(8): 999-1008, 2015 Aug.
Artículo
en Inglés
| MEDLINE | ID: mdl-25906753
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