A 16-year-old patient with Duchenne muscular dystrophy (DMD) died from acute liver failure after being treated with Sarepta Therapeutics' gene therapy, Elevidys®. This therapy aims to deliver a functional version of the dystrophin gene, which is mutated in DMD patients, using a viral vector. This approach seeks to enable dystrophin protein production, which is crucial for muscle function, potentially slowing or halting the disease's progression. This incident marks a severe case of liver injury not previously reported with this therapy. Sarepta has stated that while the treatment has a positive benefit-risk balance, it will update its prescribing information. The liver failure could have been exacerbated by a recent cytomegalovirus infection the patient had, which can damage the liver. This event has prompted discussions about gene therapies' unpredictability and investment risks.
Links:
https://www.genengnews.com/topics/genome-editing/dmd-patient-dies-after-treatment-with-sarepta-gene-therapy/
https://www.mayoclinic.org/tests-procedures/gene-therapy/about/pac-20384619
hi caitlyn! i was honestly drawn to your blog post because it talks about a case study in the beginning. i really like when blog posts are specific like that instead of only news about technologies. gene therapy is such a great treatment method and having read your blog post, i think we are in good hands that there may be good technology to get the treatment to where it needs to be to prevent certain genetic abnormalities.
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