Sarepta Therapeutics has reported the death of a second patient linked to its Duchenne muscular dystrophy gene therapy. The company is taking steps to enhance safety protocols, particularly for non-ambulatory individuals receiving the treatment. This development has raised concerns among investors, leading to a decline in Sarepta’s stock price. Other pharmaceutical companies, such as Roche, have also paused their use of similar therapies due to associated risks like liver failure. The focus now shifts to ensuring patient safety while advancing critical medical innovations.
— new from Barron’s