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FAQ

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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Antibody
Antigen
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La sindrome di Wiskott-Aldrich

Related topics:
Malattie del sangue Malattie autoimmuni Malattie rare Cellule staminali del sangue Uso attuale e potenziale

La sindrome di Wiskott-Aldrich è una malattia genetica rara che colpisce il sistema immunitario. Fondazione Telethon ha prodotto un video che spiega quali sono le cause di questa sindrome e quali sono gli effetti.

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