Organizations and Foundations
Below are organizations and foundations that have been helpful, informational, and relevant to the rare disease community and our own journey towards learning and treatment.
EveryONE Medicines delivers individualized precision therapeutics, customized to the unique pathogenic genetic mutation of patients.
Our mission is to apply the efficiency, versatility and specificity of antisense technology to charitably provide experimental antisense oligonucleotide (ASO) medicines to treat patients with nano-rare diseases.
TGen, the Translational Genomics Research Institute, part of City of Hope, is an Arizona-based, nonprofit medical research institute dedicated to conducting groundbreaking research with life-changing results. We work to unravel the genetic components of common and complex diseases, including cancer, neurological disorders, infectious disease, and rare childhood disorders. By identifying treatment options in this manner, we believe medicine becomes more rational, more precise and, well, more personal.
National Organization for Rare Diseases NORD
NORD advances practical, meaningful, and enduring change so people with rare diseases can live their fullest and best lives. Every day, we elevate care, advance research, and drive policy in a purposeful and holistic manner to lift up the rare disease community.
The Jackson Laboratory: Rare Disease Translational Center
Our team serves those with rare disease by accelerating the pre-clinical phase. Our vision is to provide patients with an efficient path from diagnosis to therapy, allowing them to live longer, healthier lives.
The EveryLife Foundation for Rare Diseases
A nonprofit, nonpartisan organization dedicated to empowering the rare disease patient community to advocate for impactful, science-driven legislation and policy that advances the equitable development of and access to lifesaving diagnoses, treatments and cures.
An advocacy organization dedicated to connecting the rare disease community. It empowers patients, builds communities while seeking to eliminate the challenges experienced by people with rare diseases.
National Center for Advancing Translational Sciences (NCATS)
This organization provides a collection of resources, Rare Diseases Resources, on topics of interest to the rare disease community. Included is information on rare disease social networks, online medical reference websites and rare disease events. NCATS is part of the National Institutes of Health (NIH).