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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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Exon Skipping Explained from Sarepta Therapeuticshttps://player.vimeo.com/video/238953537...
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Duke Children’s Neuromuscular ClinicDuke Health is committed to conducting i...
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Kennedy Krieger InstituteKennedy Krieger Institute is an internat...
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Safety, Tolerability, and Pharmacokinetics of SMT C1100, a 2-Arylbenzoxazole Utrophin Modulator, following Single- a...PURPOSE:SMT C1100 is a utrophin modulato...
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James Poysky, PhDDr. Poysky is internationally recognized...