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FAQ

What role do preclinical and clinical trials play in ensuring patient safety?
Why are some conditions more suitable for gene and cell therapies than others?
There is an approved treatment available for my condition - why can't I receive it?
How to look for approved medicines?
How are medicines developed and approved?

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Glossary

Accelerated assessment
Adeno-associated virus (AAV)
Adenovirus
Adult stem cells
Advanced Therapy Investigational Medicinal Product (ATIMP)
Advanced Therapy Medicinal Product (ATMP)
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Antibody
Antigen
Base editors

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Orphan medicines

Medicines to treat rare and severe diseases when no other satisfactory treatment is available.

Read related content in the entries on therapy classification, specifically in the entry on ATMPs and Orphan medicines of the research pathways.

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