Since our inception, NDF has delivered more than ten million dollars in grant funding to the world’s leading rare disease researchers, drug development pharmaceutical leaders, and giants in gene therapy science. Our annual grants support ongoing scientific efforts and drug development that move us rapidly forward on our path to deliver the therapeutic that will halt the progression of rare disease GNE Myopathy (GNEM).
As we proceed toward this well-deserved cure, NDF continues to fulfill its mission of enhancing the quality of life for those living with GNEM, through its uniquely broad range of patient resources and programs which educate and inform, support and connect GNEM patients around the globe.
Free Resources for GNE Myopathy Patients
GNEM Patient Huddles
NDF hosts a monthly Patient Huddle open to GNEM patients globally. The Patient Huddle is a virtual, peer-to-peer support and mentoring group focused on the cognitive health & well-being of people diagnosed with GNEM. This confidential, online support group allows patients from anywhere to discuss any topic in an open, safe, and non-judgmental forum, providing a sense of community and reliable support to our active GNEM patient community.
Speaker Series: Information for Patients of Rare Disease
NDF’s popular Speaker Series attracts the top minds and educators in the fields of rare disease, GNEM, gene therapy research, clinical healthcare, mental health and more. This patient resource facilitates a virtual forum in which renowned experts can engage directly with the global GNEM patient community. Best of all, this valuable patient resource allows for individuals living with GNEM to participate and benefit from the comfort of their homes.
Care Partner group for GNEM Patient Supporters
In June 2023, NDF launched its Care Partner Support Group, providing community and support to loved ones, family members, carers and partners of patients living with GNEM. This program facilitates patients and caregivers connecting with each other, and in some cases, cultivating rewarding lifelong friendships. Participants can share stories, ask questions, and get support from other participants. This group, which hosts meetings online, can be accessed from around the globe, and are held monthly.
$125,000.00 2023 Matching Grant
Each year, NDF sets the bar among global non-profits serving the rare disease community, with its gifting of funds that drive progress in critical research across areas such as gene therapy and pharmaceutical development. Both the research and the science enabled by NDF grants lead us ever closer to the cure that GNEM patients are awaiting. In mid 2023, NDF received a matching grant from one generous donor. Our fundraising deadline is August 31st, and we continue to inch closer to our $125,000.00 fundraising goal. This will mean an impressive $250,000.00 in cumulative grant funds, which the NDF will provide directly to ongoing scientific efforts to deliver a pre-IND package to the FDA, for the purpose of our application to proceed with further clinical trials that will ultimately deliver a human grade therapeutic to end GNEM.
NDF and How to Get Involved
The NDF (Neuromuscular Disease Foundation) is a 501(c)(3) non-profit based in Beverly Hills, California. The NDF strives to enhance the quality of lives of individuals living with and impacted by rare disease GNE Myopathy (GNEM) through education, advocacy and patient resources. We are currently accepting applications and interest from interested volunteers and/or those who can give of their time and expertise! To learn about the many ways you can help us, please click here. To contribute to our patient resources program or scientific efforts, donate here.