Liver-directed AAV-based gene therapy offers promise for people living with hemophilia B or other genetic disorders. 6,7,11–18 However, the long-term safety and efficacy of this approach remain ...

The death of a 16-year-old boy taking a gene therapy this month dealt a fresh blow to the Duchenne muscular dystrophy ...

This story was originally published on PharmaVoice. To receive daily news and insights, subscribe to our free daily PharmaVoice newsletter. The death of a 16-year-old boy taking a gene therapy ...

Sarepta Therapeutics (NASDAQ:SRPT) stock draws an Overweight rating at Wells Fargo, based on commercial prospects for ...

Acute liver injury is a known possible side effect of Elevidys and other AAV-mediated gene therapies and is highlighted in ...

Dr Tejinder Kataria, Chairperson, Radiation Oncology, Cancer Care, Medanta, Gurugram, highlights the benefits of gene therapy ...

Premium InsightsThe Hepatitis C drug market is entering a transformative phase as global health organizations, pharmaceutical ...

Despite the challenges, including what CSL CEO Paul McKenzie has described as a fragmented U.S. healthcare system, the blood ...

Acute Serious Liver Injury: Acute serious liver injury has ... Preexisting Immunity against AAVrh74: In AAV-vector based gene therapies, preexisting anti-AAV antibodies may impede transgene ...

Ultragenyx's strong revenue growth, diverse streams, and late-stage pipeline make it a standout in rare disease and gene ...

Sarepta and Roche announced a 16-year-old US patient dosed with Elevidys died of acute liver failure in March 2025.