Therapeutic developments for Duchenne muscular dystrophy

IEC Verhaart, A Aartsma-Rus - Nature Reviews Neurology, 2019 - nature.com
Duchenne muscular dystrophy (DMD) is caused by the lack of functional dystrophin protein.
Improvements in patient care and disease management have slowed down disease …

Therapeutic targeting of 3′, 5′-cyclic nucleotide phosphodiesterases: inhibition and beyond

GS Baillie, GS Tejeda, MP Kelly - Nature reviews Drug discovery, 2019 - nature.com
Phosphodiesterases (PDEs), enzymes that degrade 3′, 5′-cyclic nucleotides, are being
pursued as therapeutic targets for several diseases, including those affecting the nervous …

Contemporary cardiac issues in Duchenne muscular dystrophy

EM McNally, JR Kaltman, DW Benson, CE Canter… - Circulation, 2015 - Am Heart Assoc
Muscular Dystrophy, convened a Working Group meeting on July 2014, in Bethesda, MD, to
explore clinical and research questions related to cardiac disease in patients with DMD. As …

The pathogenesis and therapy of muscular dystrophies

S Guiraud, A Aartsma-Rus, NM Vieira… - Annual review of …, 2015 - annualreviews.org
Current molecular genomic approaches to human genetic disorders have led to an
explosion in the identification of the genes and their encoded proteins responsible for these …

Drug development progress in duchenne muscular dystrophy

J Deng, J Zhang, K Shi, Z Liu - Frontiers in Pharmacology, 2022 - frontiersin.org
Duchenne muscular dystrophy (DMD) is a severe, progressive, and incurable X-linked
disorder caused by mutations in the dystrophin gene. Patients with DMD have an absence of …

Satellite cells in muscular dystrophy–lost in polarity

NC Chang, FP Chevalier, MA Rudnicki - Trends in molecular medicine, 2016 - cell.com
Recent findings employing the mdx mouse model for Duchenne muscular dystrophy (DMD)
have revealed that muscle satellite stem cells play a direct role in contributing to disease …

Skeletal muscle quantitative nuclear magnetic resonance imaging and spectroscopy as an outcome measure for clinical trials

PG Carlier, B Marty, O Scheidegger… - Journal of …, 2016 - content.iospress.com
Recent years have seen tremendous progress towards therapy of many previously incurable
neuromuscular diseases. This new context has acted as a driving force for the development …

[HTML][HTML] Pharmacological advances for treatment in Duchenne muscular dystrophy

S Guiraud, KE Davies - Current opinion in pharmacology, 2017 - Elsevier
Highlights•Numerous pathophysiological features of DMD provide different therapeutic
avenues.•Pharmacolougical therapies target all muscles and are applicable to all DMD …

Current and emerging treatment strategies for Duchenne muscular dystrophy

JK Mah - Neuropsychiatric disease and treatment, 2016 - Taylor & Francis
Duchenne muscular dystrophy (DMD) is the most common form of muscular dystrophy in
childhood. It is caused by mutations of the DMD gene, leading to progressive muscle …

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 …