In a letter, the European Medicines Agency have asked Sarepta to place a hold on three clinical trials evaluating the gene therapy Elevidys for Duchenne muscular dystrophy.
A teenage patient on an Elevidys trial recently died after experiencing acute liver failure, a recognised potential adverse effect of this and other adeno-associated virus-based gene therapies (see BioNews 1282).
For more information, please visit Neurology Live.