Until relatively recently, boys with DMD usually did not survive much beyond their teen years. Thanks to advances in cardiac and respiratory care, life expectancy is increasing and many young adults with DMD attend college, have careers, get married, and have children. Survival into the early 30s is becoming more common than before. MDA-supported researchers are actively pursuing several exciting strategies in DMD, such as gene therapy , exon skipping , stop codon read-through and gene repair.
Human clinical trials are underway for some of these strategies. For an overview of DMD research strategies and the latest research news, see Research. On Sept. On Feb. Skip to main content. Search MDA. Search Donate. What causes DMD? What is the life expectancy in DMD?
What is the status of DMD research? Muscular dystrophy refers to a group of genetic muscle-wasting disorders. All muscular dystrophies cause muscle weakness, but life expectancy can vary dramatically between types of muscular dystrophy. While some cases may be mild and progress slowly, others can cause severe muscle weakness and disability. Some people with muscular dystrophy may have reduced life expectancy while others have a normal lifespan. Most patients with Becker muscular dystrophy live into adulthood.
With appropriate management of heart problems, they can expect to live a normal lifespan. Until recently, children with Duchenne muscular dystrophy DMD did not often live beyond their teens. However, improvements in cardiac and respiratory care mean that life expectancy is increasing , with many DMD patients reaching their 30s, and some living into their 40s and 50s. Facioscapulohumeral muscular dystrophy is a slow-progressing type of muscular dystrophy. For most patients , life expectancy is not affected by the disease.
Survival in Duchenne muscular dystrophy: improvements in life expectancy since and the impact of home nocturnal ventilation. Neuromuscul Disord. Rall S, Grimm T. Survival in Duchenne muscular dystrophy. Acta Myologica. Improvement of survival in Duchenne Muscular Dystrophy: retrospective analysis of patients. Politano L, Nigro G. Treatment of dystrophinopathic cardiomyopathy: review of the literature and personal results.
Limbgirdle muscular dystrophy type 2D gene therapy restores alphasarcoglycan and associated proteins. Ann Neurol. Disease rescue and increased lifespan in a model of cardiomyopathy and muscular dystrophy by combined AAV treatments. PLoS One. Worsening of cardiomyopathy using deflazacort in an animal model rescued by gene therapy. Combined deficiency of alpha and epsilon sarcoglycan disrupts the cardi ac dystrophin complex.
Hum Mol Genet. Theoretic applicability of antisense-mediated exon skipping for Duchenne muscular dystrophy mutations. Hum Mutat. Articles from Acta Myologica are provided here courtesy of Pacini Editore.
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