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 …
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 …
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 …
explore clinical and research questions related to cardiac disease in patients with DMD. As …
The pathogenesis and therapy of muscular dystrophies
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 …
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 …
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 …
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 …
neuromuscular diseases. This new context has acted as a driving force for the development …
[HTML][HTML] Pharmacological advances for treatment in Duchenne muscular dystrophy
Highlights•Numerous pathophysiological features of DMD provide different therapeutic
avenues.•Pharmacolougical therapies target all muscles and are applicable to all DMD …
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 …
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 …
childhood disorder that causes progressive muscle weakness and degeneration and results …