We work year-round to enhance the quality of life for those living with GNE Myopathy by funding critical research focused on treatments and a cure through The International Gene Therapy Development Program (IGTDP) and by advocacy, education and outreach.
The NDF has provided more than 9 million dollars in grant funding to facilitate research and development, moving us closer to the therapeutic that will halt the progression of GNEM.
In 2023, we are honored to have awarded grants to four impactful researchers and laboratories performing innovative research and development that will drive progress toward a brighter future for GNEM patients worldwide. The 2023 NDF grant recipients are: Julie M. Crudele, University of Washington Department of Neurology, Monkol Lek, Yale University School of Medicine, Department of Genetics, Paul T. Martin, Nationwide Children’s Hospital, Abigail Wexner Research Institute, and Stella Mitrani-Rosenbaum, Hadassah Hebrew University Medical Center, Goldyne Savad Institute of Gene Therapy.
Julie M. Crudele, University of Washington Department of Neurology: Codon optimization of GNE transgene as a method to reduce doses for GNEM AAV gene therapy
In this project, Dr. Crudele will use a codon optimization approach to reduce doses of AAV needed to achieve the same level of treatment efficacy at a lower overall risk to patients.
Monkol Lek, Yale University School of Medicine, Department of Genetics: Supporting the International Gene Therapy Development Program
The Lek lab project will support a collaboration among experts as they begin FDA approved clinical trials for GNEM using AAV-mediated gene replacement therapy.
Paul T. Martin, Nationwide Children’s Hospital, Abigail Wexner Research Institute: Studies of an inducible GNE knockout mouse model for GNE myopathy
This project will test a new mouse model for GNE myopathy (GNEM) to provide a more robust disease model for testing therapies.
Stella Mitrani-Rosenbaum, Hadassah Hebrew University Medical Center, Goldyne Savad Institute of Gene Therapy: GNE Myopathy: GneKO models for assessment of AAVGNE gene therapy
This project will develop and characterize three mice models in parallel, anticipating that at least one of these models will develop muscle pathology that could be targeted for treatment evaluation.
We are confident that these projects will move us closer to a human grade gene therapy for GNEM. Thanks to funding for these developmental projects, our goal to end GNEM is near. Through generous donations from the global GNEM community, we are able to facilitate rapid progress toward a treatment. If you would like to contribute to NDF grants, please visit https://curegnem.org/get-involved/donate/.