In addition to laying out expectations for trial progress, Pfizer offered new details on the patient death it disclosed in December, news of which triggered new concerns about the safety of gene therapy in treating Duchenne.
According to Mikael Dolsten, Pfizer’s top scientist, the patient, a 16-year-old boy with advanced Duchenne, experienced low fluid volume and cardiogenic shock, a condition in which the heart can’t pump enough blood. He was the first trial participant given an immunosuppressive drug called sirolimus as part of his treatment regimen, and had evidence of an active viral infection, Dolsten said on a conference call Tuesday.
