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Delandistrogene moxeparvovec, sold under the brand name Elevidys, is a recombinant gene therapy used for the treatment of Duchenne muscular dystrophy. [3] It is designed to deliver into the body a gene that leads to production of Elevidys micro-dystrophin that contains selected domains of the dystrophin protein present in normal muscle cells. [3]
Clinical trials for muscular dystrophy have not proven successful in generating functional improvements compared to placebo. Gains of muscle mass were small to non-existent in this population. [ 13 ] Research is ongoing on the potential use of myostatin inhibitors for motor neuron diseases like spinal muscle atrophy and amyotrophic lateral ...
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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.
“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 ...
Devices to help a person with duchenne muscular dystrophy stand are a common treatment approach. [3] Orthotic devices are used (as discussed above) and also specific standing devices such as standing frames, standing wheelchairs, and tables that have a tilt function.
Distal muscular dystrophy, also called distal myopathy, is essentially any muscle disease that preferentially affects the hands and/or feet, a much less common pattern than proximal muscle weakness. Late adult-onset type 1; Late adult-onset type 2a; Late adult-onset type 2b; Early adult-onset type 1; Early adult-onset type 2; Early adult-onset ...
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]
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