Welcome to this week’s Research Roundup. These Friday posts aim to inform our readers about the many stories that relate to animal research each week. Do you have an animal research story we should include in next week’s Research Roundup? You can send it to us via our Facebook page or through the contact form … Continue reading Research Roundup: gene therapy for inherited blindness, urea implicated as a major cause of dementia and more!
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
Stem cell hope for Duchenne muscular dystrophy
Duchenne muscular dystrophy (DMD) is an inherited disease that affects about one in every 4,000 males born in the USA. It is caused by mutations in the DMD gene that lead to the protein dystrophin being either absent or faulty, which leads to muscle cell death, progressive muscle wasting and early death, with few patients … Continue reading Stem cell hope for Duchenne muscular dystrophy