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https://www.um.edu.mt/library/oar/handle/123456789/77247| Title: | Emerging treatments for Retinitis Pigmentosa |
| Authors: | Zuccarelli, Marta Borg, John-Joseph Vella Szijj, Janis Azzopardi, Lilian M. Serracino-Inglott, Anthony |
| Keywords: | Retinitis pigmentosa Rare diseases Rare diseases -- Genetic aspects Eye -- Diseases |
| Issue Date: | 2021 |
| Publisher: | University of Malta. Department of Pharmacy |
| Citation: | Zuccarelli, M., Borg, J. J., Vella Szijj, J., Azzopardi, L. M., & Serracino-Inglott, A. (2021). Emerging treatments for Retinitis Pigmentosa. 12th World Meeting on Pharmaceutics, Biopharmaceutics and Pharmaceutical Technology. |
| Abstract: | Retinitis Pigmentosa (RP) is a class of rare-inherited retinal dystrophies which can lead to blindness and is characterised by pigment deposits predominantly present in the periphery of the retina. The hereditary pattern of RP follows a Mendelian inheritance pattern. The onset of RP typically occurs during the early teenage years with difficulties in dark adaptation, concurrent nyctalopia and reduced visual field (VF) which leads to tunnel vision and gradual reduction in central vision with a risk of blindness. Currently, no standard treatment for RP is available. One medicinal product (MP), Luxturna, which consists of voretigene neparvovec, a gene transfer vector, is authorised in the European Union (EU) and United States (US) to treat patients with inherited retinal dystrophies, caused by biallelic RPE65 mutations, and who have sufficient viable retinal cells. |
| URI: | https://www.um.edu.mt/library/oar/handle/123456789/77247 |
| Appears in Collections: | Scholarly Works - FacM&SPha |
Files in This Item:
| File | Description | Size | Format | |
|---|---|---|---|---|
| Emerging Treatements for Retinitis Pigmentosa.pdf | 230.27 kB | Adobe PDF | View/Open |
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