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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?
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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
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Marketing authorisation under exceptional circumstances

Specific approval for certain medicines targeting rare diseases or when full data collection is unethical or impossible.

Read related content in the entries on Marketing authorisation under exceptional circumstances of the research pathways.

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