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RPE65-associated inherited retinal diseases: consensus recommendations for eligibility to gene therapy.

Andrea SodiSandro BanfiFrancesco TestaMichele Della CorteIlaria PasseriniElisabetta PeloSettimio RossiFrancesca Simonellinull null
Published 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.
Keyphrases
  • gene therapy
  • clinical practice
  • systematic review
  • optical coherence tomography
  • diabetic retinopathy
  • randomized controlled trial
  • meta analyses