A nationwide clinical trial for a new type treatment that could offer help to those suffering from Duchenne Muscular Dystrophy, a devastating neuromuscular disease.
Doctors implant left ventricular assist device (LVAD) to help stop the progression of heart disease and improve the quality of life in brothers with Becker’s Muscular Dystrophy.
Scientists identified a sequence in the dystrophin gene that is essential for helping muscle tissues function, a breakthrough discovery that could lead to treatments for the deadly hereditary disease.
Recent findings by an international collaboration including IRCM researchers hold new implications for the pathogenesis of myotonic dystrophy.
RTC 13 effectively counteracts ‘nonsense’ mutation that causes disorder. Scientists at UCLA have identified a new compound that could treat certain types of genetic disorders in muscles. It is a big first step in what they hope will lead to human clinical trials for Duchenne muscular dystrophy. Duchenne muscular dystrophy, or DMD, is a degenerative [...]
Researchers from the University of Minnesota’s Lillehei Heart Institute have effectively treated muscular dystrophy in mice using human stem cells derived from a new process that – for the first time – makes the production of human muscle cells from stem cells efficient and effective. The research, published today in Cell Stem Cell, outlines the strategy [...]
The new approach could have implications for many genetic diseases. While RNA is an appealing drug target, small molecules that can actually affect its function have rarely been found. But now scientists from the Florida campus of The Scripps Research Institute have for the first time designed a series of small molecules that act against [...]
People who have the most common type of adult muscular dystrophy also have a higher risk of getting cancer, according to a paper published today in the Journal of the American Medical Association. The team found that patients who have myotonic muscular dystrophy are at increased risk primarily for four types of cancer: brain, ovary, [...]
Oxford University scientists have developed a new method for delivering complex drugs directly to the brain, a necessary step for treating diseases like Alzheimer’s, Parkinson’s, Motor Neuron Disease and Muscular Dystrophy. These diseases have largely resisted attempts to over the last 50 years develop new treatments, partly because of the difficulty of getting effective new [...]
After inserting a gene into mice that increases choline transporter and as a result increases acetylcholine at the neuromuscular junctions, the engineered mice were able to run on treadmills twice as long as controls without the inserted gene.