ISCIII
Carlos III Health Institute.
IRDiRC
International Rare Diseases Research Consortium.
FDA
US Food and Drug Administration.
EC
Clinical trial.
CIBERER
Biomedical Research Network Center for Rare Diseases.
NICE
National Institute for Health and Care Excellence
NHS
National Health Service
MH
Orphan Drug
ERDF
Spanish Federation for Rare Diseases
ER
Rare disease