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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]
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.
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 ...
The therapy, Elevidys, was granted accelerated approval in June last year to treat Duchenne muscular dystrophy, an inherited progressive muscle-wasting disorder that almost always affects young boys.
When the first gene therapy for the rare, debilitating disease Duchenne muscular dystrophy was conditionally approved earlier this year, it was hailed as a seminal moment for patients and their ...
The gene therapy will compete with Australia-based CSL Behring’s Hemgenix, a similar treatment that won FDA approval for hemophilia B in 2022. That drug has a similar list price of $3.5 million ...
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