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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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Dani Trees, PT...
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Massachusetts General HospitalThe Massachusetts General Hospital Resea...
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Duchenne Muscular Dystrophy Tissue Bank for Exon SkippingWe will utilize the Cooperative Internat...
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Adrienne Leblanc, PT, MPTI graduated in 1994 from University of S...
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Feasibility of Virtual Reality in Children With Neuromuscular Disease, Effectiveness of Virtual Reality and Biofeedb...Our study is a randomized controlled stu...
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Juliane Florence, PhDDr. Florence is the Director of clinical...
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Results of North Star Ambulatory Assessments (NSAA) in the Phase 3 Ataluren Confirmatory Trial in Patients with Nons...Objective: Examine the efficacy of atal...
