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Aide et soutien
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- Glossary
Rare Disease Resources
A rare disease is a condition that affects less than 1 in 2,000 people. It is currently estimated that there are over 7,000 rare diseases, with more being identified as science progresses. This figure may seem low, but it is important to recall that there are 6-8000 distinct rare diseases affecting individuals within Europe. This means that up to 36 million people in Europe are living with a rare disease.
This page curates resources to help readers understand these conditions, the healthcare landscape that surrounds them, and current research into gene and cell therapies that may help them.
Where should I start?
Start with our introductory guide to help you identify which resources may be most useful for you. If you are new to the topic, our basic explainers provide a helpful foundation, including an overview of what can currently be treated and what gene and cell therapy involves.
Condition-specific Factsheets
Our factsheets are clear, accessible resources designed to help patients, families, and the public understand key topics related to gene and cell therapies. These factsheets each focus on a specific condition and its biological causes, ongoing laboratory research, and the science behind emerging gene and cell therapies.
Decision-making Resources
These resources support informed decision-making about medical treatments. Together, they explain how therapies are developed and approved, why access can vary, and what factors influence whether a treatment is suitable. They also highlight key questions to consider when exploring new or emerging treatment options. Collectively, these materials provide context to increase understanding of treatment pathways, availability, and the broader healthcare landscape.
External Resources
We also curate external resources from trusted institutes and organisations, including educational content, explainers, videos and reports.
