Analysis of the Gene Therapies Authorized by the United States Food and Drug Administration and the European Medicines Agency.

Buthainah GhanemEnrique Seoane-VazquezLawrence BrownRosa Rodriguez-Monguio

Published in: Medical care (2023)

Gene therapy is used to treat incurable diseases that affect only a small number of patients (orphan diseases). Based on this, they are approved by the EMA and FDA with insufficient clinical evidence to ensure safety and efficacy, in addition to the high cost.

Keyphrases

drug administration
gene therapy
end stage renal disease
newly diagnosed
chronic kidney disease
prognostic factors
genome wide
copy number
gene expression
patient reported