[HTML][HTML] Cardiac pathophysiology and the future of cardiac therapies in Duchenne muscular dystrophy

TA Meyers, DW Townsend - International journal of molecular sciences, 2019 - mdpi.com
Duchenne muscular dystrophy (DMD) is a devastating disease featuring skeletal muscle
wasting, respiratory insufficiency, and cardiomyopathy. Historically, respiratory failure has …

[HTML][HTML] Standard of care versus new-wave corticosteroids in the treatment of Duchenne muscular dystrophy: Can we do better?

S Kourakis, CA Timpani, DG Campelj, P Hafner… - Orphanet Journal of …, 2021 - Springer
Background Pharmacological corticosteroid therapy is the standard of care in Duchenne
Muscular Dystrophy (DMD) that aims to control symptoms and slow disease progression …

Efficacy and safety of vamorolone vs placebo and prednisone among boys with Duchenne muscular dystrophy: a randomized clinical trial

M Guglieri, PR Clemens, SJ Perlman, EC Smith… - JAMA …, 2022 - jamanetwork.com
Importance Corticosteroidal anti-inflammatory drugs are widely prescribed but long-term use
shows adverse effects that detract from patient quality of life. Objective To determine if …

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 …

[HTML][HTML] Muscle and cardiac therapeutic strategies for Duchenne muscular dystrophy: past, present, and future

A Łoboda, J Dulak - Pharmacological Reports, 2020 - Springer
Background Duchenne muscular dystrophy (DMD) is a severe X-linked neuromuscular
childhood disorder that causes progressive muscle weakness and degeneration and results …

[HTML][HTML] Duchenne muscular dystrophy: Disease mechanism and therapeutic strategies

A Bez Batti Angulski, N Hosny, H Cohen… - Frontiers in …, 2023 - frontiersin.org
Duchenne muscular dystrophy (DMD) is a severe, progressive, and ultimately fatal disease
of skeletal muscle wasting, respiratory insufficiency, and cardiomyopathy. The identification …

Role of mineralocorticoid receptor antagonists in kidney diseases

V Patel, A Joharapurkar, M Jain - Drug development research, 2021 - Wiley Online Library
Mineralocorticoid receptor (MR) antagonists, for example, spironolactone and eplerenone,
are in clinical use to treat hypertension. Increasing evidence suggests that mineralocorticoid …

Developing DMD therapeutics: a review of the effectiveness of small molecules, stop-codon readthrough, dystrophin gene replacement, and exon-skipping therapies

O Sheikh, T Yokota - Expert opinion on investigational drugs, 2021 - Taylor & Francis
Introduction Duchenne muscular dystrophy (DMD) is an X-linked recessive disorder caused
by mutations in the dystrophin (DMD) gene. Most patients die from respiratory failure or …

Vamorolone trial in Duchenne muscular dystrophy shows dose-related improvement of muscle function

EP Hoffman, BD Schwartz, LJ Mengle-Gaw, EC Smith… - Neurology, 2019 - AAN Enterprises
Objective To study vamorolone, a first-in-class steroidal anti-inflammatory drug, in Duchenne
muscular dystrophy (DMD). Methods An open-label, multiple-ascending-dose study of …

Myostatin/activin receptor ligands in muscle and the development status of attenuating drugs

BD Rodgers, CW Ward - Endocrine Reviews, 2022 - academic.oup.com
Muscle wasting disease indications are among the most debilitating and often deadly
noncommunicable disease states. As a comorbidity, muscle wasting is associated with …