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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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Catabasis Quarterly: Updates on edasalonexent and clinical trialsHappy New Year! As 2019 kicks off, our P...
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Dystrophin-deficient dogs with reduced myostatin have unequal muscle growth and greater joint contracturesBackground: Myostatin (Mstn) is a negat...
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Matthew Wood, PhDMatthew Wood is Professor of Neuroscienc...
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Santhera’s Raxone® receives first positive EAMS scientific opinion from UK’s MHRA in Duchenne muscular ...The aim of the EAMS is to provide patien...
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Safety, Tolerability, and Pharmacokinetics of Single and Multiple Doses of HT-100 in Duchenne Muscular DystrophyThe main purpose of this study is to tes...
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A 2-Part Study to Assess the Safety and Tolerability, pk, Effects on Histology and Some Clinical Parameters of Givin...This is a 2-part, phase 2 study to asses...
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Phase 2a Extension Study of Ataluren (PTC124) in Duchenne Muscular Dystrophy (DMD)Duchenne muscular dystrophy (DMD) is a g...
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Nancy Shepard, PTNancy Shepard, PT, works at The MDA/Neur...
