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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)
Advertising 
Antibody
Antigen
Base editors

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Compassionate use

The exceptional possibility for individual patients to access unauthorized medicines outside of a clinical trial. 

 

Read related content in the entries on therapy classification of the research pathways.

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