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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).
rareRelated
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Induced Pluripotent Stem Cells for Duchenne Muscular Dystrophy Modeling and TherapyDuchenne muscular dystrophy (DMD) is an ...
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Preliminary Data and Intention to Dose Escalate in IGNITE DMDToday we announced our intention to move...
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Myocardial T1 and T2 Mapping in Duchenne muscular dystrophy: characterization of late gadolinium enhancementBackground: In the current era, cardiov...
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FibroGen Receives Orphan Drug Designation from the U.S. FDA For Pamrevlumab for the Treatment of Duchenne Muscular D...FibroGen, Inc., a leading biopharmaceu...
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CRISPR-SKIP: Programmable gene splicing with single base editorsCRISPR gene editing has revolutionized b...
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Neuromuscular & Neurometabolic Clinic at McMaster Childen’s HospitalNeuromuscular & Neurometabolic Clini...
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Stefan Spinty, MDDr. Spinty studied medicine at the Unive...
