Kenny Walter

The professor of medicine at Duke University School of Medicine previously served as commissioner in 2016.

Patients with CDI also had more severe acute graft vs. host disease than those without CDI.

FIX activity was similar in participants without and with pre-existing neutralizing antibodies.

Following 2 unexpected serious adverse events, the trial was put on clinical hold as of February 2021.

Researchers observed 20-25% frequencies of this 4.9 kb deletion in HSPCs prior to xenotransplantation and at 17 weeks post-engraftment.

Multiple myeloma is currently incurable with many patients experiencing relapses.

The investigators found the primary efficacy endpoints occurred in 20 evaluable patients (87%).

FLT201 is a combination of AAVS3 capsid and a liver-specific promotor to drive the expression of GCasevar85.

The 1 patient who did not received immunomodulatory agents demonstrated the highest level of FVIII activity.

The researchers unexpectedly found sustained visual improvement in both eyes, despite only 1 eye being treated with AAV.

Minimal fat infiltration was observed on MR images from the SRP-9001 arm compared to participants from the natural history cohort.

No serious adverse events reported through 24 weeks of a 52 week trial.

During the annual ASH meeting, investigators present phase 1 data from a new dose-climbing trial.