welcome to DuchenneXchange
- a positively charged Duchenne muscular dystrophy community.- join today!
- log in
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
-
Novel rod domain duplication in dystrophin resulting in X-linked dilated cardiomyopathyBackground: X-linked dilated cardiomyop...
-
Sarepta Therapeutics Announces Positive Expression Results from the Casimersen (SRP-4045) Arm of the ESSENCE StudySarepta Therapeutics, Inc., the leader i...
-
Ronald Cohn, MD, FACMGRonald Cohn joined The Hospital for Sick...
-
Study of Ataluren in Previously Treated Participants With Nonsense Mutation Dystrophinopathy (nmDBMD)The objective of this study is to assess...
-
Hillary Zebberman, MSWHillary Zebberman is a Licensed Clinical...
-
Basil Darras, MDDr. Darras serves as an expert in the De...
-
Catabasis Pharmaceuticals Initiates Phase 3 PolarisDMD Clinical Trial For Edasalonexent In Duchenne Muscular Dystrop...Catabasis Pharmaceuticals, Inc., a clini...
