RPE65-associated inherited retinal diseases: consensus recommendations for eligibility to gene therapy.
Andrea SodiSandro BanfiFrancesco TestaMichele Della CorteIlaria PasseriniElisabetta PeloSettimio RossiFrancesca Simonellinull nullPublished in: Orphanet journal of rare diseases (2021)
Consensus guidelines were developed to guide paediatricians and general ophthalmologists to arrive at the correct diagnosis of RPE65-associated IRD and make informed clinical decisions regarding eligibility for a gene therapy approach to RPE65-associated IRD. The guidelines aim to ensure the best outcome for the patient, based on expert opinion, the published literature, and practical experience in the field of IRDs.