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Inhibiting NF-κB improves heart function in a mouse model of Duchenne muscular dystrophy
Duchenne muscular dystrophy (DMD) is a devastating genetic disease that impairs cardiac and skeletal muscle development. People with DMD gradually lose ambulation in childhood, acquire respiratory and heart failure in young adulthood and succumb to the disease by their mid-thirties. Until recently, there has been no effective treatment for the characteristic muscle-wasting progression of this disease. Provisional FDA approval of the first DMD therapy (eteplirsen) and improved disease management strategies have extended the life span of DMD patients and expanded the field of DMD research into later-stage outcomes such as cardiomyopathy (heart failure).
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Nathalie Goemans, MD, PhDDr. Nathalie Goemans is the head of the ...
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Effect of pulmonary function on right heart function in Duchenne muscular dystrophy patientsBackground Duchenne muscular dystrophy ...
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Pediatric Aquatic Therapy (Pool Therapy) for Muscular Dystrophy Patientshttps://www.youtube.com/watch?v=YAqnQnZe...
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Rally for RyanRyan, a 13 year old boy, who lives in th...
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Safety and Tolerability of WVE-210201 in Patients With Duchenne Muscular DystrophyThis is a Phase 1, double-blind, placebo...
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The Children’s Hospital Los Angeles Neuromuscular ClinicThe Children’s Hospital Los Angeles Ne...
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Massachusetts General HospitalThe Massachusetts General Hospital Resea...
