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“Duchenne muscular dystrophy (DMD) is one of the most severe forms of inherited muscular dystrophies,” the National Library of Medicine says. “Mutations in the dystrophin gene lead to ...
Another source reports Duchenne muscular dystrophy being a rare disease and having an occurrence of 7.1 per 100,000 male births. [9] A number of sources referenced in this article indicate an occurrence of 6 per 100,000. [10] Duchenne muscular dystrophy is the most common type of muscular dystrophy, [3] with a median life expectancy of 27–31 ...
Successful treatment by way of exon skipping could lead to a mostly functional dystrophin protein, and create a phenotype similar to the less severe Becker muscular dystrophy (BMD). [1] [5] In the case of Duchenne muscular dystrophy, the protein that becomes compromised is dystrophin. [5]
The diagnosis of muscular dystrophy is based on the results of muscle biopsy, increased creatine phosphokinase (CpK3), electromyography, and genetic testing. A physical examination and the patient's medical history will help the doctor determine the type of muscular dystrophy.
The US Food and Drug Administration has given the green light for the first gene therapy that treats a rare form of muscular dystrophy to be used in most people who have the disease and a certain ...
The advantage of using these cell types for therapy in muscle diseases is that they can be systemically delivered, autonomously migrating to the site of injury. Particularly successful recently has been the delivery of mesoangioblast cells into the Golden Retriever dog model of Duchenne muscular dystrophy, which effectively cured the disease. [24]
Furthermore, individuals who have mutations in both copies of the myostatin gene (popularly—but inaccurately—called the "Hercules gene") have significantly more muscle mass and are stronger than normal. There is hope that studies into myostatin may have therapeutic application in treating muscle wasting diseases such as muscular dystrophy. [12]
Myoblast transfer in the treatment of Duchenne's Muscular Dystrophy (DMD): [16] an in vivo technique to replace dystrophin, a skeletal muscle protein which is deficient in patients with DMD; myoblasts fuse with muscle fibers and contribute their nuclei which then replace deficient gene products in the host nuclei