Promising clinical trial result for exon skipping in Duchenne Muscular Dystrophy

Last Monday the Muscular Dystrophy Campaign announced that the antisense oligonucleotide drug AVI-4658 had performed well in its second clinical trial in 19 boys with Duchenne Muscular Dystrophy (DMD). DMD is one of the most common hereditary disorders affecting the skeletomuscular system, and which results in progressive muscle degeneration, difficulty in walking and breathing, and … Continue reading Promising clinical trial result for exon skipping in Duchenne Muscular Dystrophy

Progress towards a cure for Duchenne Muscular Dystrophy

Duchenne muscular dystrophy (DMD) is a severe inherited muscular dystrophy that causes progressive muscle degeneration which eventually leads to loss of the ability to use muscles and death, and every year tens thousands of children are born afflicted with the disease. It is caused by mutations in the DMD gene that encodes dystrophin, a protein … Continue reading Progress towards a cure for Duchenne Muscular Dystrophy