{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,23]],"date-time":"2026-10-23T20:56:15Z","timestamp":1792788975973,"version":"4.5.0"},"reference-count":27,"publisher":"MDPI AG","issue":"1","license":[{"start":{"date-parts":[[2024,1,17]],"date-time":"2024-01-17T00:00:00Z","timestamp":1705449600000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"funder":[{"name":"Novartis Pharma AG"}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["Biomolecules"],"abstract":"<jats:p>Voretigene neparvovec (VN) is the first available gene therapy for patients with biallelic RPE65-mediated inherited retinal dystrophy who have sufficient viable retinal cells. PERCEIVE is an ongoing, post-authorization, prospective, multicenter, registry-based observational study and is the largest study assessing the real-world, long-term safety and effectiveness of VN. Here, we present the outcomes of 103 patients treated with VN according to local prescribing information. The mean (SD) age was 19.5 (10.85) years, 52 (50.5%) were female, and the mean (SD) duration of the follow up was 0.8 (0.64) years (maximum: 2.3 years). Thirty-five patients (34%) experienced ocular treatment-emergent adverse events (TEAEs), most frequently related to chorioretinal atrophy (n = 13 [12.6%]). Eighteen patients (17.5%; 24 eyes [13.1%]) experienced ocular TEAEs of special interest, including intraocular inflammation and\/or infection related to the procedure (n = 7). The mean (SD) changes from baseline in full-field light-sensitivity threshold testing (white light) at month 1, month 6, year 1, and year 2 were \u221216.59 (13.48) dB (51 eyes), \u221218.24 (14.62) dB (42 eyes), \u221215.84 (14.10) dB (10 eyes), and \u221213.67 (22.62) dB (13 eyes), respectively. The change in visual acuity from baseline was not clinically significant. Overall, the outcomes of the PERCEIVE study are consistent with the findings of VN pivotal clinical trials.<\/jats:p>","DOI":"10.3390\/biom14010122","type":"journal-article","created":{"date-parts":[[2024,1,17]],"date-time":"2024-01-17T04:16:56Z","timestamp":1705465016000},"page":"122","update-policy":"https:\/\/doi.org\/10.3390\/mdpi_crossmark_policy","source":"Crossref","is-referenced-by-count":71,"title":["Real-World Safety and Effectiveness of Voretigene Neparvovec: Results up to 2 Years from the Prospective, Registry-Based PERCEIVE Study"],"prefix":"10.3390","volume":"14","author":[{"given":"M. Dominik","family":"Fischer","sequence":"first","affiliation":[{"name":"Centre for Ophthalmology, University of T\u00fcbingen, 72076 T\u00fcbingen, Germany"},{"name":"Oxford Eye Hospital, Oxford University NHS Foundation Trust, Oxford OX3 9DU, UK"},{"name":"Nuffield Laboratory of Ophthalmology, Nuffield Department of Clinical Neurosciences, University of Oxford, Oxford OX3 9DU, UK"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Francesca","family":"Simonelli","sequence":"additional","affiliation":[{"name":"Eye Clinic, Multidisciplinary Department of Medical, Surgical and Dental Sciences, University of Campania Luigi Vanvitelli, Via S. Pansini, 5, 80131 Napoli, Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-0970-8211","authenticated-orcid":false,"given":"Jayashree","family":"Sahni","sequence":"additional","affiliation":[{"name":"Novartis Pharma AG, 4056 Basel, Switzerland"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Frank G.","family":"Holz","sequence":"additional","affiliation":[{"name":"Department of Ophthalmology, University of Bonn, Ernst-Abbe-Stra\u00dfe 2, 53127 Bonn, Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Rainer","family":"Maier","sequence":"additional","affiliation":[{"name":"Novartis Pharma AG, 4056 Basel, Switzerland"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Christina","family":"Fasser","sequence":"additional","affiliation":[{"name":"Retina International, D02 TW98 Dublin, Ireland; Retina Suisse, 8005 Z\u00fcrich, Switzerland"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Andrea","family":"Suhner","sequence":"additional","affiliation":[{"name":"Novartis Pharma AG, 4056 Basel, Switzerland"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Daniel P.","family":"Stiehl","sequence":"additional","affiliation":[{"name":"Novartis Pharma AG, 4056 Basel, Switzerland"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Bee","family":"Chen","sequence":"additional","affiliation":[{"name":"Novartis Pharmaceutical Corporation, East Hanover, NJ 7936, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-0698-5309","authenticated-orcid":false,"given":"Isabelle","family":"Audo","sequence":"additional","affiliation":[{"name":"Sorbonne Universit\u00e9, INSERM, CNRS, Institut de la Vision, 75012 Paris, France"},{"name":"15\u201320 H\u00f4pital National de la Vision, National Rare Disease Center REFERET, INSERM-DGOS CIC1423, 75012 Paris, France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-9899-2081","authenticated-orcid":false,"given":"Bart P.","family":"Leroy","sequence":"additional","affiliation":[{"name":"Department of Ophthalmology & Center for Medical Genetics Ghent, Ghent University & Ghent University Hospital, 9000 Ghent, Belgium"},{"name":"Children\u2019s Hospital of Philadelphia, Philadelphia, PA 19104, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"1968","published-online":{"date-parts":[[2024,1,17]]},"reference":[{"key":"ref_1","doi-asserted-by":"crossref","unstructured":"Olivares-Gonz\u00e1lez, L., Velasco, S., Campillo, I., and Rodrigo, R. 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