Publication alert! Best practices for Empa use in GSD1b

We are excited to announce that this very important publication is now fully available online: Treatment recommendations for glycogen storage disease type IB- associated neutropenia and neutrophil dysfunction with empagliflozin: Consensus from an international workshop.
This publication is the result of an international workshop, fully funded by Sophies Hope Foundation, that brought together over a dozen physicians, researchers, dieticians and patients/caregivers from around the world to create consensus-based recommendation on the use of empa in GSD1b. Until now, there have been no established guidelines for this off-label drug use, which means that some patients struggled to get the drug prescribed and clinicians had less guidance to use in caring for patients. This is not a new struggle in the world of rare disease, but a problem we wanted to remedy for 1b patients on empa. We are very proud of this collaboration and hope it will benefit 1b patients across the globe. Huge thanks to several of our scientific advisors, including Dr. Saskia Wortmann, Dr. Sarah Grunert and Dr. Terry Derks for leading this effort!
Link to publication is HERE. Remember, you can also find other publications on GSD1b in our Published Literature Database right here on the website!

Share this post