Elevidys now carries a black box warning for acute liver injury and failure, limiting its use to ambulatory patients aged four and older. The FDA's actions follow fatal liver failure reports in ...
SRPT's Elevidys gets a sharply narrowed FDA label with new liver safety warnings that reshape how the gene therapy can be used.
Key Takeaways The FDA now limits Elevidys to walking boys ages 4 and up after two teens died from liver failureThe therapy ...
The therapeutic landscape for hepatocellular carcinoma (HCC) has been revolutionized by the advent of molecular targeted therapies and immune ...
Sarepta must also run a post-marketing study for Elevidys to better assess the risk of serious liver injury in patients dosed ...
Dr. Christopher Kelly: A biomarker is a biological substance that can be objectively measured and tracked to provide ...
By reactivating a long-lost gene, researchers were able to lower uric acid levels and stop damaging fat accumulation in human ...
The FDA added a boxed warning to delandistrogene moxeparvovec (Elevidys) gene therapy for Duchenne muscular dystrophy and changed the indication of the drug after reports of fatal liver injury emerged ...
The U.S. Food and Drug Administration said on Friday it approved new labeling for Sarepta Therapeutics' gene therapy Elevidys ...
This multi-target blood test "offers the potential for improved patient outcomes through earlier HCC detection," John said ...
The FDA is requiring the manufacturer to conduct a postmarketing observational study to further assess the risk of serious liver injury. The study will enroll approximately 200 patients with DMD and ...
Prime Medicine, Inc. ( PRME) Discusses Liver Disease Franchise Strategy With Focus on Wilson Disease November 12, 2025 8:00 AM EST ...
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