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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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Volker Straub, MD, PhDProfessor Volker Straub was founding joi...
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Ataluren: An Overview of Clinical Trial Results in Nonsense Mutation Duchenne Muscular DystrophyObjective: Provide an overview of atalu...
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Mary Colvin, PhDDr. Mary Colvin is a staff neuropsycholo...
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Parent Project Muscular Dystrophy Designates Children’s National Health System, a Certified Duchenne Care Cent...Parent Project Muscular Dystrophy (PPM...
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Utrophin modulators to treat Duchenne muscular dystrophy (DMD): Phase 1b Clinical Trial Results of SMT C1100Objective: To assess the safety, tolera...
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Ronald Smith, MBBS, FRCPCDr. Ronald Smith is a Developmental Pedi...
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A Phase 3 Trial of Pamrevlumab or Placebo in Combination With Systemic Corticosteroids, in Subjects With Non-ambulat...To evaluate the efficacy and safety of p...
