The Rare Heroes
While awareness is the crucial first step for rare diseases, taking action is what makes a real difference. On this page, I have curated a list of trusted organisations working tirelessly in the rare disease space. Some focus on specific conditions, while others represent thousands worldwide. Use this guide to find safe, reliable ways to contribute, whether through donations, volunteering, or simply spreading the word.
Global & Umbrella Organisations
| Organisation | Description |
|---|---|
| EURORDIS | A pan-European alliance representing 1,000+ patient organisations in 70+ countries, advocating for people with rare diseases and influencing policy worldwide. |
| National Organization for Rare Disorders (NORD) | A U.S.-based non-profit that provides education, advocacy, research funding, and direct patient/family support for those living with rare diseases. |
| Rare Diseases International (RDI) | A global network uniting national and regional alliances to strengthen advocacy and policy at the international level. |
| Orphanet | A comprehensive database offering medical and scientific information about rare diseases and orphan drugs, widely used by doctors, researchers, and patients. |
| Global Genes | A leading U.S.-based rare disease advocacy group that connects patients, caregivers, and researchers while raising awareness and providing educational resources. |
| Rarewell | A Hong Kong-based non-profit supporting individuals with rare diseases through awareness campaigns, patient stories, and a directory of patient groups. |
| APARDO | The Asia Pacific Alliance of Rare Disease Organisations, focusing on advocacy, policy development, and support for rare disease communities in the Asia-Pacific region. |
| GARD (NIH) | The Genetic and Rare Diseases Information Centre, part of the U.S. National Institutes of Health, providing information and resources for rare diseases. |
Research & Medical Support Charities
| Organisation | Description |
|---|---|
| Muscular Dystrophy Association (MDA) | A U.S. nonprofit that supports research, clinical care, and community programs for people affected by neuromuscular diseases. |
| Action Medical Research | A UK charity funding medical research into rare and childhood diseases, including premature birth, disability, and rare genetic conditions. |
| Foundation for Rare Diseases (Fondation Maladies Rares) | A French foundation supporting scientific research across many rare diseases. |
| AFM-Téléthon | A major French charity funding neuromuscular disease research, patient services, and advocacy. Known for its large annual fundraising telethon. |
Disease-Specific Foundations
| Organisation | Description |
|---|---|
| DEBRA (Dystrophic Epidermolysis Bullosa Research Association) | An international network supporting people living with epidermolysis bullosa (EB). DEBRA funds research, provides patient services, and advocates for better care. |
| Foundation Fighting Blindness | Funds research on retinal degenerative diseases such as retinitis pigmentosa, macular degeneration, and Usher syndrome. |
| World Duchenne Organization | A global federation focused on advocacy, awareness, and research for Duchenne muscular dystrophy. |
| International Pompe Association (IPA) | An international organisation dedicated to raising awareness and supporting research for Pompe disease. |
| Myeloma Patients Europe (MPE) | A network of patient organisations dedicated to improving outcomes for people affected by multiple myeloma and AL amyloidosis. The organisation supports research, advocacy, and education to advance treatment access and quality of care across Europe. |
| Malaysia Lysosomal Diseases Association (MLDA) | A Malaysian non-profit advocating for patients with lysosomal storage disorders, providing education and support. |
National Alliances
| Organisation | Description |
|---|---|
| Genetic Alliance Australia | An Australian organisation supporting individuals and families affected by genetic and rare diseases, focusing on advocacy and policy. |
| Rare Voices Australia | A national voice for Australians living with rare diseases, advocating for improved access to care and support. |
| Fabry Australia | An Australian organisation providing support and information for individuals affected by Fabry disease. |
| Genetic Alliance UK | A national network supporting individuals and families affected by genetic, rare, and undiagnosed conditions. Runs Rare Disease UK, a national policy campaign. |
How to Choose a Trustworthy Charity
| Check Charity Watchdogs: Look up organisations on Charity Navigator, Guidestar, or your country’s charity regulator. Look for Transparency: Reputable charities usually publish annual reports and financial statements. Research Their Impact: See if the organisation highlights concrete achievements (e.g., research funded, patient services provided, policy changes influenced). Watch for Red Flags: Avoid charities that make vague promises, do not list board members or contact info, or pressure for donations. Consider Local vs. Global: Some people prefer supporting international umbrella groups, while others want to donate to disease-specific or local patient organisations. |
Disclaimer
The organisations on this list are provided as a resource. Please note that I am not affiliated with any of these groups and receive no financial benefit from their inclusion. While each has been chosen for its reputation, I strongly encourage you to perform your own due diligence before donating to ensure the organisation aligns with your personal goals.
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