Sarepta Announces that Screening and Enrollment are Underway in ENDEAVOR Cohort 8 to Evaluate Enhanced Immunosuppression Regimen as Part of ELEVIDYS Gene Therapy for Non-Ambulant Individuals with Duchenne
View original at finance.yahoo.comSarepta Announces that Screening and Enrollment are Underway in ENDEAVOR Cohort 8 to Evaluate Enhanced Immunosuppression Regimen as Part of ELEVIDYS Gene Therapy for Non-Ambulant Individuals with Duchenne - Approximately 25 non-ambulatory participants will receive sirolimus as part of the regimen in Cohort 8 of the END…
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Sarepta is on an urgent mission to engineer precision genetic medicine for rare diseases that devastate lives and cut futures short
60% confidenceIn non-ambulatory patients treated with ELEVIDYS, acute liver failure with fatal outcome has occurred in the clinical and post-marketing settings
60% confidenceThe enhanced immunosuppressive regimen with sirolimus is designed to mitigate the risk of acute liver injury and acute liver failure associated with AAV gene therapy in non-ambulatory patients
60% confidenceThe approach for Cohort 8 is based on preclinical data and shaped by real-world clinical experience, including guidance from independent specialists in Duchenne and liver health
60% confidenceCohort 8 of ENDEAVOR is expected to build on dystrophin expression data while deepening understanding of safety profile in older patients with more advanced disease
60% confidenceIndividuals with non-ambulatory Duchenne face profound unmet need and fewer treatment options
60% confidenceAcute serious liver injury, including life-threatening and fatal acute liver failure, has occurred with ELEVIDYS, particularly in non-ambulatory patients
60% confidence