A patient with Duchenne muscular dystrophy has died following treatment with Elevidys, a gene therapy developed by Sarepta Therapeutics, the company announced in a safety update. The news sent the company’s stock down about 23 percent in morning trading on March 18.
“We are profoundly saddened to share that a young man with Duchenne muscular dystrophy has passed away following treatment with ELEVIDYS, having suffered acute liver failure,” a company report read.
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“Patient safety and well-being are Sarepta’s top priority. We continue to gather and analyze the information from this event,” the company added.
Sarepta Therapeutics, a major player in genetic medicine, stated that while acute liver injury is a known risk associated with Elevidys and other “AAV-mediated gene therapies,” acute liver failure leading to death had not previously been reported among the more than 800 patients treated with the therapy.
In its statement, Sarepta noted that the patient had also recently contracted cytomegalovirus (CMV), which the treating physician identified as a possible contributing factor to his condition. CMV can affect the liver and lead to CMV hepatitis, the company said, potentially compounding the risks associated with gene therapy. […]
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