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Muscle-targeted gene therapy reverses rare muscular dystrophy in mice

Gene therapy methods that specifically target muscle may reverse the symptoms of a rare form of muscular dystrophy, according to new research in mice conducted by medical geneticists at Duke University Medical Center. Infants born with the inherited muscular disorder called Pompe disease usually die before they reach the age of two. The researchers also said their approach of targeting corrective...

Chemical 'band-aid' prevents heart failure in mice with muscular dystrophy

A common chemical used in the manufacturing and pharmaceutical industries can repair damage to cardiac muscle cell membranes and prevent heart failure in mice with the genetic mutation that causes Duchenne muscular dystrophy, according to scientists at the University of Michigan Medical School. The mutation in the dystrophin gene causes the progressive deterioration of skeletal muscles see...

No small feat: First ever gene therapy success for muscular dystrophy achieved

Researchers from the University of Pittsburgh report the first study to achieve success with gene therapy for the treatment of congenital muscular dystrophy (CMD) in mice, demonstrating that the formidable scientific challenges that have cast doubt on gene therapy ever being feasible for children with muscular dystrophy can be overcome. Moreover, their results, published in this week's online edi...

Gene therapy reverses genetic mutation responsible for heart failure in muscular dystrophy

University of Pittsburgh investigators have for the first time used gene therapy to successfully treat heart failure and other degenerative muscle problems in an animal model that is genetically susceptible to a human muscular dystrophy. Reporting in the Oct. 25 edition of the journal Circulation, the authors say that this is the first successful attempt to deliver a therapeutic gene throughout t...

Gene therapy for muscular dystrophy fixes frail muscle cells in animal model, Stanford study finds

A new gene therapy technique that has shown promise in skin disease and hemophilia might one day be useful for treating muscular dystrophy, according to a new study by researchers at Stanford University School of Medicine. In the study, scheduled to be published online in the Proceedings of the National Academy of Sciences the week of Jan. 2, the researchers used gene therapy to introduce...

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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...

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PTC Therapeutics Announces Additional Positive Interim Phase 2 Results of PTC124 in Duchenne Muscular Dystrophy

Data Presented at 59th American Academy of Neurology Annual Meeting BOSTON and SOUTH PLAINFIELD, N.J., May 04, 2007 /PRNewswire/ --PTC Therapeutics, Inc. (PTC), a biopharmaceutical company focusedon the discovery and development of small-molecule drugs targetingpost-transcriptional control processes, today announced positiveinterim data from a Phase 2 clinical trial of PTC124 in patientsw...

Insmed Announces Promising Results From IPLEX Phase II Myotonic Muscular Dystrophy Clinical Study

RICHMOND, Va.--(BUSINESS WIRE)--May 3, 2007 - Insmed Inc.(Nasdaq:INSM) today announced positive results from a Phase IIinvestigator-sponsored study of the company's drug, IPLEX(TM), inpatients with myotonic muscular dystrophy (DM1). Preliminary results of the clinical study, being conducted atthe University of Rochester School of Medicine and Dentistry,showed that six months of treatment...
dystrophy at Medicine Dictionary

Reflex Sympathetic Dystrophy

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Myotonic Dystrophy

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Muscular dystrophy

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Leukodystrophy

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Adrenoleukodystrophy

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