Sarepta therapeutics is seeking to enroll 25 non-ambulant individuals with Duchenne in a clinical trial evaluating the use of sirolimus as part of an enhanced immunosuppressive regimen prior to and after Elevidys treatment. This data will aim to determine whether sirolimus treatment can help reduce the risk of acute liver injury with AAV gene therapy. Commercial use of Elevidys for non-ambulatory individuals with Duchenne was paused last year after cases of acute liver failure resulting in death occurred. Additional information about this trial cohort can be found on clinicaltrials.gov (NCT04626674)
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