• FAQs
  • Glossary
Close

FAQs

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?

See all FAQs

Close

Glossary

Adeno-associated virus (AAV)
Adenovirus
Adult stem cells
Antibody
Antigen
Base editors
Cas9 nuclease
Chimeric Antigen Receptor (CAR)
Chromosome
Clinical trial

See full Glossary

European Joint Programme on Rare Diseases (EJP RD) Resources

Bringing over 130 institutions from 35 countries, the European Joint Programme on Rare Diseases (EJP RD) aims to create an effective, comprehensive, and sustainable rare diseases research ecosystem.

Key information and resources provided on the site include:

EJP RD_toolbox

EJP RD Innovation Management Toolbox

EJP RD's reference library of resources in rare disease translational medicine

Learn more
EJP RD logo

EJP RD Trainings

EJP RD's Training and Education programmes

Learn more

Additional information and resources by EJP RD:

Back to Key Pathway Resources

External links

Back to top
Did you find the content on this page useful? If not, you can leave us a message so we can improve Send us your thoughts