The application of Aptamer in biomarker discovery

Y Li, WW Tam, Y Yu, Z Zhuo, Z Xue, C Tsang, X Qiao… - Biomarker …, 2023 - Springer
Biomarkers are detectable molecules that can reflect specific physiological states of cells,
organs, and organisms and therefore be regarded as indicators for specific diseases. And …

Efficacy and safety of vamorolone in Duchenne muscular dystrophy: a 30-month nonrandomized controlled open-label extension trial

JK Mah, PR Clemens, M Guglieri, EC Smith… - JAMA network …, 2022 - jamanetwork.com
Importance Vamorolone is a synthetic steroidal drug with potent anti-inflammatory
properties. Initial open-label, multiple ascending dose-finding studies of vamorolone among …

Vamorolone: first approval

SJ Keam - Drugs, 2024 - Springer
Vamorolone (AGAMREE®) is an oral, selective, dissociative corticosteroid developed by
ReveraGen BioPharma and Santhera Pharmaceuticals for the treatment of patients with …

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

EC Smith, LS Conklin, EP Hoffman, PR Clemens… - PLoS …, 2020 - journals.plos.org
Background Treatment with corticosteroids is recommended for Duchenne muscular
dystrophy (DMD) patients to slow the progression of weakness. However, chronic …

[HTML][HTML] Dimethyl fumarate modulates the dystrophic disease program following short-term treatment

CA Timpani, S Kourakis, DA Debruin, DG Campelj… - JCI insight, 2023 - ncbi.nlm.nih.gov
New medicines are urgently required to treat the fatal neuromuscular disease Duchenne
muscular dystrophy (DMD). Dimethyl fumarate (DMF) is a potent immunomodulatory small …

[HTML][HTML] Causes of clinical variability in Duchenne and Becker muscular dystrophies and implications for exon skipping therapies

EP Hoffman - Acta Myologica, 2020 - ncbi.nlm.nih.gov
Becker muscular dystrophy is caused by mutations in the DMD gene that permit significant
residual dystrophin protein expression in patient muscle. This is in contrast to DMD gene …

Multiomic characterization of disease progression in mice lacking dystrophin

M Signorelli, R Tsonaka, A Aartsma-Rus, P Spitali - PLoS One, 2023 - journals.plos.org
Duchenne muscular dystrophy (DMD) is caused by genetic mutations leading to lack of
dystrophin in skeletal muscle. A better understanding of how objective biomarkers for DMD …

Identification of auxiliary biomarkers and description of the immune microenvironmental characteristics in duchenne muscular dystrophy by bioinformatical analysis …

X Han, J Han, N Wang, G Ji, R Guo, J Li… - Frontiers in …, 2022 - frontiersin.org
Background Duchenne muscular dystrophy (DMD) is a genetic muscle disorder
characterized by progressive muscle wasting associated with persistent inflammation. In this …

A blood biomarker for Duchenne muscular dystrophy shows that oxidation state of albumin correlates with protein oxidation and damage in mdx muscle

BA Al-Mshhdani, MD Grounds, PG Arthur, JR Terrill - Antioxidants, 2021 - mdpi.com
Duchenne muscular dystrophy (DMD) is a severe X-linked muscle wasting disease with no
cure. While the precise mechanisms of progressive dystropathology remain unclear …

Considering the promise of vamorolone for treating Duchenne muscular dystrophy

MD Grounds, EM Lloyd - Journal of Neuromuscular Diseases, 2023 - content.iospress.com
This commentary provides an independent consideration of data related to the drug
vamorolone (VBP15) as an alternative steroid proposed for treatment of Duchenne muscular …