For the past 10 years, we have funded over $1.3 million in clinical research globally, resulting in greater interest from principal investigators and industry alike.
We are spearheading a formal consortium of key researchers who have agreed to collaborate, convene annual, and share data through a single database to expedite a cure. Members of our scientific collaboration include: Ultragenyx Pharmaceuticals, PerkinElmer, The Open Medicine Institute, Emory University, UCLA, UC Irvine, JScreen, The Jain Foundation, Ohio State University, Nationwide Children’s Hospital, and the National Institutes of Health (NIH).
We maintain one of the largest GNE Myopathy patient registries in the world to ensure that the community is organized and trial-ready and to collect data needed for better disease understanding.
We encourage patients to register for natural history studies.
Our commitment to the intersection of technology and biology has led to the launch of an investigation with the FDA for gene therapy.
We advocate for additional gene therapy funding estimated at $25 million with third-party biotech firms, high net worth stakeholders, and non-governmental grant makers.