Patient advocacy groups
We help you find the most appropriate patient organizations worldwide to guide you through rare diseases and to find individual assistance.
Rare Diseases International (RDI)
Rare Diseases International is a global alliance of individuals living with any rare disease. It aims to bring together national and regional rare disease patient organizations and create a strong common voice to advocate for rare disease as an international public health priority. The website includes background info about the groups activities and details of member organizations.
Washington DC, USA
The Genetic Alliance is a large nonprofit advocacy organization. They have a number of tools and programs to promote access to information, research and services for patients and families affected by genetic diseases. In addition, the Genetic alliance has a network of more than 1200 disease-specific advocacy organizations as well as universities, companies and government agencies which they can help you to engage with.
Amyloidosis - The International Society of Amyloidosis (ISA)
Germany, Italy, UK, USA,
The ISA is an organization of scientists and doctors engaged in research or practice concerning the disease amyloidosis. It contains basic information about the disease and links to research and medical centers around the world involved in amyloidosis treatment.
This is a large online network for the cystic fibrosis international community. The site hosts an active online cystic fibrosis forum plus information on clinical trials, gene therapy, testing, associations, research and events. You can also sign up for a newsletter.
Cystic Fibrosis Worldwide (CFW)
Started in 2002, CFW is a nonprofit organization that has provided a variety of programs to aid CF communities. The website has some information on CF and details of many other nonprofit CF organizations around the world.
Cure Cystinosis International Registry (CCIR)
A registration site for patients diagnosed or with symptoms of cystinosis to assist clinicians, pharmaceutical companies and other researchers developing new clinical trials for cystinosis. Information, news and comprehensive information about clinical trials including an overview of their purpose and recent progress.
Fabry International Network
FIN is a network of patient organizations from over 45 countries around the world. FIN aims to collaborate, communicate and promote best practices to support those affected by Fabry disease. The website has in depth information about the disease, diagnosis, its symptoms and how to manage these, plus recent articles published on the subject. You can find details on the network’s members and access news and sign up for their newsletter.
Glycoprotein Storage Diseases - The International Advocate (ISMRD)
ISMRD is an international consortium of nonprofit organizations who advocates for families and patients affected by one of the following disorders: Alpha-Mannosidosis, Aspartylglucosaminuria, Beta-Mannosidosis, Fucosidosis, Galactosialidosis, Sialidosis (Mucolipidosis I), Mucolipidosis alpha/beta, Mucolipidosis III Gamma, Schindler Disease.
Leukodystrophy Alliance - WLA
The World Leukodystrophy Alliance (WLA) is a consortium of non-profit organizations all of which work to improve access to quick diagnosis, and the best therapies for leukodystrophy patients. You can find some information about leukodystrophies on their site, details of all the member organizations as well as info on an annual conference, and other meetings and fundraising events.
Niemann-Pick Disease Alliance
An active consortium of patient organizations dealing with NPD. You can find a lot of information and links on their website. They curate a global registry of all NPD patients to which you can enroll, and they promote sharing of knowledge, resources and skills between patient groups. The NPD alliance organizes campaigns to improve diagnosis, publishes regular news (including updates on the latest trials) including events pages.
Austria, France, Germany, Italy, Switzerland, UK
A global alliance of patient organizations focused on research towards improving the outcome for those with polycystic kidney disease. The website contains info on the disease, a Place where you can sign up for news updates and links to associated organizations as well as PKD research and trials databases.
International Pompe Association (IPA)
The IPA is an umbrella organization for Pompe disease patient groups worldwide. It aims to coordinate activities and share experience and knowledge across the different groups. It has a comprehensive website with a good number of links about the association, its members, information brochures and updates on recent clinical trials.
Tuberous Sclerosis Complex International (TSCi)
The TSCi is a consortium of organizations supporting those affected by tuberous sclerosis complex. As well as promoting awareness, the group is a platform through which to share information, and it co-funds research projects. The website contains many links to information about the disease including upcoming meetings, research updates, and details of member organizations.