European regulators have blocked approval of a Duchenne muscular dystrophy gene therapy, Elevidys, amid growing safety concerns worldwide. Japan and Brazil have also halted distribution of the treatment while investigations continue. The US is not currently using the therapy due to increasing scrutiny. Developed by Sarepta Therapeutics, Elevidys has faced challenges after a boy in Brazil died following its use, despite his doctor attributing the cause to another factor.
Source: https://www.statnews.com/2025/07/25/elevidys-duchenne-europe-gene-therapy-sarepta-roche