The following research papers can be downloaded as useful resources for information on identifying and referring patients.
EFFICACY AND SAFETY OF VORETIGENE NEPARVOVEC (AAV2-HRPE65V2) IN PATIENTS WITH RPE65-MEDIATED INHERITED RETINAL DYSTROPHY: A RANDOMISED, CONTROLLED, OPEN-LABEL, PHASE 3 TRIAL
This paper was published in The Lancet in 2017.
EFFICACY, SAFETY, AND DURABILITY OF VORETIGENE NEPARVOVEC-RZYL IN RPE65 MUTATION–ASSOCIATED INHERITED RETINAL DYSTROPHY
This paper was published in Ophthalmology in 2019.
THE NATURAL HISTORY OF INHERITED RETINAL DYSTROPHY DUE TO BIALLELIC MUTATIONS IN THE RPE65 GENE
This paper was published in the American Journal of Ophthalmology in 2019.
LONGITUDINAL STUDY OF RPE65-ASSOCIATED INHERITED RETINAL DEGENERATIONS
This paper was published in Retina in 2020.
IRD, inherited retinal dystrophy; RPE, retinal pigment epithelium.
Indication: Luxturna is indicated for the treatment of adult and paediatric patients with vision loss due to inherited retinal dystrophy caused by confirmed biallelic RPE65 mutations and who have sufficient viable retinal cells.1
- Luxturna SmPC, 2020.