Real-World Safety and Effectiveness of Voretigene Neparvovec: Results up to 2 Years from the Prospective, Registry-Based PERCEIVE Study

Real-World Safety and Effectiveness of Voretigene Neparvovec: Results up to 2 Years from the Prospective, Registry-Based PERCEIVE Study

Voretigene neparvovec (VN) is the first available gene therapy for patients with biallelic <i>RPE65</i>-mediated inherited retinal dystrophy who have sufficient viable retinal cells. PERCEIVE is an ongoing, post-authorization, prospective, multicenter, registry-based observational study...

Full description

Bibliographic Details
Main Authors: M. Dominik Fischer, Francesca Simonelli, Jayashree Sahni, Frank G. Holz, Rainer Maier, Christina Fasser, Andrea Suhner, Daniel P. Stiehl, Bee Chen, Isabelle Audo, Bart P. Leroy
Format: Article
Language:English
Published: MDPI AG 2024-01-01
Series:Biomolecules
Subjects:
effectiveness
real world
<i>RPE65</i>-associated inherited retinal dystrophies
safety
voretigene neparvovec
Online Access:https://www.mdpi.com/2218-273X/14/1/122

Internet

https://www.mdpi.com/2218-273X/14/1/122

Similar Items