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 …

Mitochondria as a drug target in ischemic heart disease and cardiomyopathy

AM Walters, GA Porter Jr, PS Brookes - Circulation research, 2012 - Am Heart Assoc
Ischemic heart disease is a significant cause of morbidity and mortality in Western society.
Although interventions, such as thrombolysis and percutaneous coronary intervention, have …

Late‐life rapamycin treatment reverses age‐related heart dysfunction

JM Flynn, MN O'Leary, CA Zambataro… - Aging cell, 2013 - Wiley Online Library
Rapamycin has been shown to extend lifespan in numerous model organisms including
mice, with the most dramatic longevity effects reported in females. However, little is known …

Early myopathy in Duchenne muscular dystrophy is associated with elevated mitochondrial H2O2 emission during impaired oxidative phosphorylation

MC Hughes, SV Ramos, PC Turnbull… - Journal of cachexia …, 2019 - Wiley Online Library
Background Muscle wasting and weakness in Duchenne muscular dystrophy (DMD) causes
severe locomotor limitations and early death due in part to respiratory muscle failure. Given …

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 …

[HTML][HTML] Idebenone attenuates ferroptosis by inhibiting excessive autophagy via the ROS-AMPK-mTOR pathway to preserve cardiac function after myocardial …

D Li, G Zhang, Z Wang, J Guo, Y Liu, Y Lu, Z Qin… - European journal of …, 2023 - Elsevier
Cardiovascular diseases (CVDs) are the leading causes of mortality worldwide. As a type of
CVDs, myocardial infarction (MI) induces ischemia hypoxia, which leads to excessive …

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 …

Cardiomyopathy of Duchenne muscular dystrophy: current understanding and future directions

CF Spurney - Muscle & nerve, 2011 - Wiley Online Library
Duchenne muscular dystrophy (DMD) is the most common and severe form of muscular
dystrophy and occurs in 1 in 3500 male births. Improved survival due to improvements in …

Assessment and management of respiratory function in patients with Duchenne muscular dystrophy: current and emerging options

A LoMauro, MG D'Angelo, A Aliverti - Therapeutics and clinical risk …, 2015 - Taylor & Francis
Duchenne muscular dystrophy (DMD) is an X-linked myopathy resulting in progressive
weakness and wasting of all the striated muscles including the respiratory muscles. The …

Mammalian animal models for Duchenne muscular dystrophy

R Willmann, S Possekel, J Dubach-Powell… - Neuromuscular …, 2009 - Elsevier
Duchenne muscular dystrophy (DMD) is a fatal neuromuscular disease that affects boys and
leads to early death. In the quest for new treatments that improve the quality of life and in the …