Duchenne muscular dystrophy (DMD) is a progressive muscle disorder that causes the loss of both muscle function and independence. DMD is perhaps the most prevalent of the muscular dystrophies and is the most common lethal genetic disorder diagnosed during childhood today. Each year, approximately 20,000 children worldwide are born with DMD (one of every 3,500 male children).
Injecting a gene responsible for making a specific protein into a mouse that's used as a model for muscular dystrophy can lead to long-term improvements in the animal's muscle size and strength, a new study shows.
An experimental drug that has proven effective in treating muscular dystrophy also works for cystic fibrosis, according to researchers at the University of Alabama at Birmingham (UAB).
Block the action of a protein that normally regulates muscle mass, and watch your muscles grow.
Scientists in Texas have been able to restore muscle function in mice with muscular dystrophy by using embryonic stem cells.
Using embryonic stem cells from mice, UT Southwestern Medical Center researchers have prompted the growth of healthy - and more importantly, functioning - muscle cells in mice afflicted with a human model of Duchenne muscular dystrophy.
One of the nation's pre-eminent genetic researchers, Eric Hoffman, PhD, of Children's Research Institute at Children's National Medical Center, predicts that in relatively short order, medicine's next innovation--individualized molecular therapies--will have the unprecedented ability to treat muscular dystrophies, and other disorders.
Scientists report that adult stem cells isolated from humans with muscular dystrophy can be genetically corrected and used to induce functional improvement when transplanted into a mouse model of the disease.
An Australian scientist has been awarded a $1 million research grant to develop innovative gene therapy tools for muscle-related diseases.
Heart disease is the leading cause of death in the United States and greatly affects the quality and length of life for individuals with specific forms of muscular dystrophy.
PTC124, an oral medication that changes the way muscle cells interpret genetic information, holds promise as a treatment for some patients with Duchenne muscular dystrophy (DMD), the Muscular Dystrophy Association ( http://www.mda.org/ ) announced today.
Researchers have discovered the first microRNAs - tiny bits of code that regulate gene activity - linked to each of 10 major degenerative muscular disorders, opening doors to new treatments and a better biological understanding of these debilitating, poorly understood, often untreatable diseases.
Children with neuromuscular diseases such as muscular dystrophy, may be at risk for suboptimal bone density due to low levels of vitamin D.
For the first time, scientists at Children's Hospital of Pittsburgh of UPMC have discovered a unique population of adult stem cells derived from human muscle that could be used to treat muscle injuries and diseases such as heart attack and muscular dystrophy.
A major grant of approximately $10 million from the National Institutes of Health will help investigators at Columbus Children's Research Institute (CCRI) at Columbus Children's Hospital learn more about strategies to treat muscular dystrophy, potentially leading to two new gene therapy approaches.
Researchers at the University of Pennsylvania School of Medicine report how the gene for utrophin, which codes for a protein very similar to dystrophin, the defective protein in Duchenne muscular dystrophy (DMD), puts the brakes on its own expression in muscle cells, thereby suggesting a new target for treatment.
Scientists will only make real breakthroughs in children's medicine if they include children in research programmes as well as adults, according to a leading paediatric expert.
During vigorous exercise, heart muscle cells take a beating. In fact, some of those cells rupture, and if not for a repair process capable of resealing cell membranes, those cells would die and cause heart damage (cardiomyopathy).
A new genetic test targeting the most common types of muscular dystrophy--those caused by mutations in the dystrophin gene--is far quicker with greater accuracy and sensitivity than existing tests.
Boys with Duchenne muscular dystrophy were able to walk on their own for a longer period of time and reduce their risk of scoliosis as a result of receiving daily steroid treatments for several years, according to a study published in the May 8, 2007, issue of Neurology, the scientific journal of the American Academy of Neurology.
Using a new type of drug that targets a specific genetic defect, researchers at the University of Pennsylvania School of Medicine, along with colleagues at PTC Therapeutics Inc. and the University of Massachusetts Medical School, have for the first time demonstrated restoration of muscle function in a mouse model of Duchenne's muscular dystrophy (DMD).