It's been a big year for GNEM.

In spite of the recent disappointing news about the phase 3 clinical trials on Sialic Acid *, we are eternally grateful to Ultragenyx, their scientists, and for their true leadership. Special thanks to Ultragenyx's Associate Director, Patient Advocacy, Kim Mooney , for her tireless encouragement, and to CEO, Dr. Emil Kakkis for his inspiration. Without Dr. Kakkis' generosity (both personal and professional) many of our programs would not have been possible. We thank them sincerely on behalf of all our patients and constituents for their years of kinship.
* Click to read Dr. Kakkis' letter to patients .

Post-Symposium Science updates:
NDF Gene Therapy-2017
The reality of Gene Therapy is upon us, and NDF is finalizing its 1st step; the Pre-IND process. NDF's partner, Dr. Jerry Mendell of Nationwide Children’s Hospital in Columbus, Ohio, presented this and necessary next steps at our recent Symposium dinner. You can watch his presentation, here .

NDF Collaboration with the NIH- 2017
We are delighted to announce that o ur Board of Directors has voted to support the extension of NIH’s phase 2 trial of ManNAc. This agreement was reached in early October, and will be funded immediately in Q4.

Click below to learn more about the ManNAc trial, or for detailed questions contact us here directly.