Dystrophin-deficient cardiomyopathy

F Kamdar, DJ Garry - Journal of the American College of Cardiology, 2016 - jacc.org
Dystrophinopathies are a group of distinct neuromuscular diseases that result from
mutations in the structural cytoskeletal Dystrophin gene. Dystrophinopathies include …

The golden retriever model of Duchenne muscular dystrophy

JN Kornegay - Skeletal muscle, 2017 - Springer
Duchenne muscular dystrophy (DMD) is an X-linked disease caused by mutations in the
DMD gene and loss of the protein dystrophin. The absence of dystrophin leads to myofiber …

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 …

Circumferential strain analysis identifies strata of cardiomyopathy in Duchenne muscular dystrophy: a cardiac magnetic resonance tagging study

KN Hor, J Wansapura, LW Markham, W Mazur… - Journal of the American …, 2009 - jacc.org
Objectives: This study sought to evaluate the natural history of occult cardiac dysfunction in
Duchenne muscular dystrophy (DMD). Background: Duchenne muscular dystrophy is …

Oral corticosteroids and onset of cardiomyopathy in Duchenne muscular dystrophy

BJ Barber, JG Andrews, Z Lu, NA West… - The Journal of …, 2013 - Elsevier
Objective To estimate the age when cardiomyopathy develops in boys with Duchenne
muscular dystrophy (DMD) and to analyze the effect of corticosteroid treatment on the age of …

Sildenafil reverses cardiac dysfunction in the mdx mouse model of Duchenne muscular dystrophy

CM Adamo, DF Dai, JM Percival… - Proceedings of the …, 2010 - National Acad Sciences
Duchenne muscular dystrophy (DMD) is a progressive and fatal genetic disorder of muscle
degeneration. Patients with DMD lack expression of the protein dystrophin as a result of …

Long-term improvement in mdx cardiomyopathy after therapy with peptide-conjugated morpholino oligomers

N Jearawiriyapaisarn, HM Moulton… - Cardiovascular …, 2010 - academic.oup.com
Aims The cardiomyopathy found in Duchenne muscular dystrophy (DMD) is responsible for
death due to heart failure in∼ 30% of patients and additionally contributes to many DMD …

Chronic administration of membrane sealant prevents severe cardiac injury and ventricular dilatation in dystrophic dogs

DW Townsend, I Turner, S Yasuda… - The Journal of …, 2010 - Am Soc Clin Investig
Duchenne muscular dystrophy (DMD) is a fatal disease of striated muscle deterioration
caused by lack of the cytoskeletal protein dystrophin. Dystrophin deficiency causes muscle …

Early regional myocardial dysfunction in young patients with Duchenne muscular dystrophy

L Mertens, J Ganame, P Claus, N Goemans… - Journal of the American …, 2008 - Elsevier
BACKGROUND: In young patients (aged< 12 years) with Duchenne muscular dystrophy
(DMD), cardiac systolic function is generally described to be within the normal range. Recent …

Absence of full-length dystrophin impairs normal maturation and contraction of cardiomyocytes derived from human-induced pluripotent stem cells

JM Pioner, X Guan, JM Klaiman… - Cardiovascular …, 2020 - academic.oup.com
Aims Heart failure invariably affects patients with various forms of muscular dystrophy (MD),
but the onset and molecular sequelae of altered structure and function resulting from full …