Sarepta Therapeutics is a biotechnology company focused on pioneering innovative gene therapies for the treatment of rare genetic diseases, particularly those affecting muscle disorders
The company is dedicated to developing therapies that can enable patients with conditions like Duchenne Muscular Dystrophy to achieve improved outcomes and quality of life. With a strong emphasis on research and development, Sarepta utilizes advanced techniques such as RNA-targeted therapies and gene editing to combat genetic disorders at their source. Through its work, the company aims to transform the landscape of genetic medicine and provide hope for individuals and families affected by these challenging diseases.
Shares of biotech company Sarepta Therapeutics (NASDAQSRPT)
fell 24.9% in the pre-market session after the company reported the death of a Duchenne muscular dystrophy patient following treatment with its gene therapy, ELEVIDYS. The patient reportedly suffered acute liver failure, a known risk of AAV-mediated gene therapies. However, Sarepta suggested that a recent cytomegalovirus (CMV) infection might have worsened the condition. Despite this, the company maintained that ELEVIDYS still had a favorable benefit-risk profile. It planned to update the prescribing information and inform regulators, clinical investigators, and prescribing physicians of the incident.
Sarepta shares fell after reporting a patient death linked to Elevidys. The case involved acute liver failure, with a CMV infection as a potential factor.
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Biotech company Sarepta Therapeutics (NASDAQSRPT) announced better-than-expected revenue in Q4 CY2024, with sales up 65.9% year on year to $658.4 million. Its non-GAAP profit of $1.90 per share was 7.8% below analysts’ consensus estimates.
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Sarepta's EMBARK study confirms Elevidys offers sustained Duchenne treatment benefits, including motor function improvement and minimal muscle pathology progression.
