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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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The Muscular Dystrophy Association Center at Johns HopkinsThe Muscular Dystrophy Association Cente...
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Study of Eteplirsen in Young Patients With DMD Amenable to Exon 51 SkippingThis is a multicenter, open-label, dose-...
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Experts review current and potential treatments for Duchenne muscular dystrophyExperts provided an overview of the many...
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Russell J. Butterfield, MD, PhDDr. Russell Butterfield is an Associate ...
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A Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of a Single Dose of SRP-5051 in Patients With Duc...The purpose of this study is to evaluate...
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Therapeutic advances in muscular dystrophyThe muscular dystrophies comprise a hete...
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Eric Hoffman, PhDDr. Hoffman has worked on Duchenne Muscu...
