Founded in 2006 by Gila Michael, NDF is a registered 501(c)(3) public charity, and the world's leading foundation in funding research that supports the search for a cure for GNE Myopathy (also known as HIBM). Research is just one of the four programs within the NDF. Click here to learn more about these programs.The organization is governed by its rotating Board of Directors, managed by its Executive Director, and supported by volunteer committees, including a distinguished Scientific Advisory Committee.
Our Mission Statement:
The Neuromuscular Disease Foundation's (NDF) mission is to enhance the quality of the lives of people living with GNE Myopathy (also known as HIBM) through advocacy, education, outreach and funding critical research focused on treatments and a cure.
Our 2019 Impact:
- Funded Hadassah Medical Center with a 2-year grant for creation of animal models. Their successful zebra fish model—as a direct result of this funding— is the first reliable animal model developed that can be used in clinical testing and can ascertain the efficacy of the approach. Dr. Mitrani-Rosenbaum’s lab is also developing a mouse model due in 2020.
- Funding of Lek Lab at Yale University biomarker and CRISPR studies. The results obtained from the Lek Lab will allow us to determine the feasibility of using this approach in clinical trials.
- Funded Dr. Kelly Crowe at Mount St. Joseph/ Nationwide Children’s Hospital’s project using sialylation-sensitive lectins to identify biomarkers of the disease which can then be used to assess the therapeutic potential of therapies such as gene therapy. This important study may also help get valid outcome measures for future clinical trials.
- Completed 1st phase of Whole Genomic Sequencing in 40 samples tested at PerkinElmer and Yale, with some additional assistance from the NIH. This helps identify any modifiers and allows the medical community to better understand the reasons that patients show such diversity in the progressions of the disease.
- Provided gift funds to NIH for critical human resources support for their GNEM studies. This supports late stage clinical trials for ManNAc to develop outcome measures to be used for FDA approval for ManNAc, as well as for any future treatments currently in development.
Advocacy - NDF Events
- Hosted & produced symposia in Philadelphia and Tel Aviv
- Co-sponsored patient events in Israel, India, Côte d’Ivoire, Palo Alto, and New York
- Sponsored multiple NDF Ambassadors awareness and friend-raising events
- Created NDF All Star Awards contest Facilitated monthly patient HUDDLES
- Hosted quarterly Certified Patient Advocacy (CPA) meetings
- GNE Biobank Progress with special guest, Dr. Monkol Lek
- NIH Update on Phase 2 of ManNAc with special guest, Dr. Nuria Carrillo
- Coping with Disappointment & Grief with special guest, Dr. Gretchen Kubacky
2019 by the Numbers
26% - Growth in NDF patient registry
50 states - Now registered as a charitable organization across the US
522 - Attendees at our sold-out Gala
220 - Symposia/Patient Day attendees
18 - Core advisors
210 - Ambassadors’ Events attendees
11 - Board members
38 - Scientific minds engaged with GNEM & NDF
3 - Employees
$ 3,187,000 511 $ 22,000
donated & pledged donors raised on social media