dystrophy

Stem cells offer hope for muscular dystrophy

Italian researchers said on Thursday they were combining two new and experimental therapies -- using stem cells and gene therapy -- to try to treat muscular dystrophy. Their early work has shown some success in mice but they warned any real progress is years away. Writing in the journal Science, the team lead by Dr. Giulio Cossu of the Stem Cell Research Institute in Milan and the Univer...

Gene Therapy Gives Hope For Patients With Muscular Dystrophy

Researchers say their recent discovery could be the first step in the path for a treatment for humans with muscular dystrophy. They say for the first time they have found a successful way to deliver gene therapy // to all of the muscles in a mouse. Gene therapy for muscular dystrophy has been a hopeful treatment idea. However, finding a delivery method for the whole body has been a majo...

The Use Of Oral Steroids For Muscular Dystrophy Questioned

Treating children who have Duchenne muscular dystrophy (DMD) with the oral steroid prednisone is probably a good idea say researchers // despite the side effects of the drug . Most patients with this form of muscular dystrophy die in their late teens or early 20s from respiratory failure or heart problems. Despite the beneficial effects of the drug, however, many have questioned whether i...

Different Drug May Help Children With Duchenne Muscular Dystrophy

According to a new study, the drug perindopril used in early treatment of children with Duchenne muscular dystrophy (DMD) may// delay the onset and progress of left ventricle dysfunction. DMD is one of the more common forms of muscular dystrophies that are characterized by muscle enlargement and rapid degeneration of muscles in early life. This disease is linked with the X-chromosomes a...

Treatment for heart damage in muscular dystrophy may be possible

Common chemical used in manufacturing industries may be able to repair heart damages// and prevent heart failures of people suffering from muscular dystrophy. Duchenne muscular dystrophy (DMD) is one of the more common forms of muscular dystrophies that are characterized by muscle enlargement and rapid degeneration of muscles in early life. This disease is linked with the X-chromosomes...

Hope For Patients With Muscular Dystrophy

Researchers are now saying that the gene known as “mighty mouse” might be beneficial in human cells as well. Studies on this gene arose when a German neurologist , stumbled // on a newborn who appeared to have especially well-developed muscles. Further tests revealed the child had a mutation in his myostatin gene that effectively kept his body from producing the myostatin protein, which...

Gene Therapy Overcomes Heart Failure In Muscular Dystrophy

Researchers at the University of Pittsburgh have successfully treated heart failure in an animal model that is genetically //prone to develop a human muscular dystrophy. The article detailing this study is published in the Oct. 25 edition of the journal Circulation. The authors say that this is the first time that a gene therapy has been used in a therapeutic way. Lead author, Xiao Xiao...

Importance Of Early Diagnosis And Treatment For Muscular Dystrophy Patients

Cardiac disease is the major cause of mortality in patients with muscular dystrophy and is present in most boys with Duchenne muscular dystrophy and approximately 70 % of those with Becker muscular dystrophy. // These are the two common forms of muscular dystrophy caused by defects in a gene called dystrophin. Both are frequently associated with dilated cardiomyopathy (DCM), heart failure...

Muscular Dystrophy May Be Cured By Gene Therapy

Muscular dystrophy may also be cured by a gene therapy technique which has been promising where hemophilia and skin diseases are concerned, according to the Stanford University School of Medicine researchers // . The gene dystrophin was introduced into mice by the researchers as a part of the experiment. According to Thomas Rando, MD, there is nothing new in using gene therapy for treatin...

First Gene Therapy Human Trial For Muscular Dystrophy In Progress

The first clinical trial of gene therapy is underway for muscular dystrophy in the United States. // The clinical trial for Duchenne muscular dystrophy (DMD) tests the safety and effectiveness of a therapy that was developed over two decades by scientists at the University of North Carolina at Chapel Hill's School of Medicine and the University of Pittsburgh. The trial wa...

Muscular Dystrophy Reversed In Lab Mice

A new study appearing in Nature Genetics says that researchers have found a way to reverse muscular dystrophy (MD) in laboratory mice. This discovery kindles hope// that a cure for the human form of the disease may be found. The University of Virginia researchers said that the therapy used by them could fully restore heart and skeletal muscle function. "The results represent the firs...

Toxic Strand Of RNA Causes Myotonic Muscular dystrophy

Studies have indicated that myotonic dystrophy in adults, which is the most common type of myotonic dystrophy could be due to a toxic RNA. // Doctors at the University of Virginia Health System have shown for the first time that by getting rid of poisonous RNA (ribonucleic acid) in muscle cells could result in the reversing myotonic dystrophy. Statistics show that around 40,...

Anti-cancer drug may treat muscular dystrophy

Recent studies on mice reveal that a drug, being tested as an anticancer agent to treat breast cancer, supports recovery from muscular dystrophy//, a disease that has no cure. Duchene muscular dystrophy is a disease characterized by progressive weakness and degeneration of the muscles that control movement. According to earlier studies, the functional decline of "dystrophic" muscles ca...

Over-exercising Not Good for Muscular Dystrophy Patients

People suffering from muscular dystrophy should avoid over exercising, say experts.// Pain or a heavy feeling in the arms or legs after exercise can be caused due to training of body muscles, according to the Germany Professional Association of Neurologists. Muscular dystrophy is a genetic disease that causes progressive muscle weakness and over 30 forms of the condition hav...

Common BP Drug Treats Muscular Dystrophy

Johns Hopkins Researchers have shown that a drug commonly used to lower blood pressure reverses muscle wasting// in genetically engineered mice with Marfan syndrome and also prevents muscle degeneration in mice with Duchenne muscular dystrophy. The results are reported online this week at Nature Medicine. In 2006, a team led by Harry "Hal" Dietz, M.D., discovered that treating...

Switching Genes to Overdrive Improves Muscular Dystrophy Symptoms in Mice

BOSTON -- Scientists at Dana-Farber Cancer Institute have shown in a laboratory study that revving up a crucial set of muscle genes counteracts// the damage caused by a form of muscular dystrophy. Reporting in the April 1 issue of Genes and Development, the researchers demonstrated that manipulating a genetic molecular switch increased the genes’ activity in the muscles of mice with D...

Scientists Explore ‘muscle’ in a New Drug to Assuage Muscular Dystrophy Symptos

Muscular dystrophy is a debilitating cluster of genetic disorders that weaken the muscles to such an extent, ultimately crushing the victim’s mobility. The disorder// hampers the victim’s quality of life, while also reducing the lifespan. Unfortunately, the cure for this disorder still remains a burning ambition for many scientists. Duchenne Muscular Dystrophy or DMD is the most notori...

Daily Steroids Helpful for Boys Down With Muscular Dystrophy

Receiving daily steroid treatment has been found beneficial for boys affected by Duchenne muscular dystrophy. A new study has found that daily intake of steroids helps them to walk on their own for a longer period of time and reduce their risk of scoliosis. Duchenne muscular dystrophy occurs in one in 3,500 boys. Symptoms start in early childhood and rapidly progress with most boys lo...

New Target for Muscular Dystrophy Drug Therapy Discovered

Researchers at the University of Pennsylvania School of Medicine have discovered a new target for the muscular dystrophy drug therapy . The researchers have found 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. The producti...