Advocacy organizations can help you link with others who have rare diseases and provide valuable information as well as additional resources.
EveryLife Foundation is 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, treatment and cures.
Global Genes is 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.
Lysosomal Disease Network
Although individually rare “orphan” conditions, the lysosomal diseases collectively affect 1 in 6,000 individuals and are responsible for a significant disability and disease burden. These diseases have become a test-bed for some of the most innovative and advanced experimental treatments, including treatment agents designed to cross the blood-brain barrier.
The rarity of each lysosomal disease means that no single medical research center has an opportunity to see the entire spectrum, or to acquire sufficient patient numbers to adequately test new therapies. The combined and integrated efforts of the NIH-funded Lysosomal Disease Network focus limited resources into a network of centers with expertise in one or more of these diseases in order to solve major challenges in diagnosis, disease management, and therapy. Solutions to these problems will have direct impact on patients suffering from lysosomal diseases, and important implications for medical practice.
National Organization for Rare Diseases (NORD)
The National Organization for Rare Disorders (NORD) is a unique federation of voluntary health organizations dedicated to helping people with rare “orphan” 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).