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FAQs

What is gene and cell therapy?
What are ATMPs, and where do they fit into gene and cell therapy?
Why are animal models used when testing the safety of a new therapy?
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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Directory of Patient Organisations for Rare Diseases

Related topics:
Blood disorders Immune Conditions Rare Diseases

Orphanet have created a database of patient organisations for patients living with rare conditions, to help you connect with support.

Directory of Patient Organisations

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Orphanet: The portal for rare diseases and orphan drugs

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Person with questions

Do you have a decision to make around your own healthcare, or the healthcare of a loved one?

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What conditions can currently be treated using gene and cell therapy?

Factsheet
Related Topics Cell Therapy Current and Potential Uses Factsheets Gene Therapy Unproven Treatments
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