Useful links – The portal for rare diseases and orphan drugs. – Searchable database which provides patients, family members and the public with information about current ongoing clinical research studies.


Disease InfoSearch provides information about diseases and their related support and advocacy networks. It began as a feature of Genetic Alliance’s website, and this expansion improves both navigation and the number of information sources included.


NIH (US)How to find a disease specialist? These resources may be able to assist in your search for a healthcare professional with experience in a particular condition.


The French Muscular Dystrophy Association (AFM-Téléthon) is composed of patients and their families who are affected by a genetic, rare and progressive illness that leads to heavy handicaps: neuromuscular diseases.


List of National Alliances for Rare Diseases. National Alliances federate patient organisations from a wide range of diseases within their particular country. National rare disease alliances are stronger than national single disease patient groups (when addressing common issues) because they represent a high number of patients and can speak with one voice.


The European Medicines Agency (EMA) is a decentralised agency of the European Union, located in London. The Agency is responsible for the scientific evaluation of medicines developed by pharmaceutical companies for use in the European Union.


NIH (US)Rare Disease Resources. A collection of resources on topics of interest to the rare disease community, including rare disease social networks, online medical reference Web sites, rare disease events, and more.


 is a free database accessing primarily the MEDLINE database of references and abstracts on life sciences and biomedical topics. The United States National Library of Medicine (NLM) at the National Institutes of Health maintains the database.


The European Union Committee of Experts on Rare Diseases is charged with aiding the European Commission with the preparation and implementation of Community activities in the field of rare diseases.


The International Rare Disease Research Consortium (IRDiRC) teams up researchers and organizations investing in rare diseases research in order to achieve two main objectives by the year 2020, namely to deliver 200 new therapies for rare diseases and means to diagnose most rare diseases.

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