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https://www.um.edu.mt/library/oar/handle/123456789/48525| Title: | Developing safe and effective medicinal products to treat Leber hereditary optic neuropathy. Clinical and regulatory challenges |
| Authors: | Zuccarelli, Marta Borg, John-Joseph Serracino-Inglott, Anthony Vella, Janis |
| Keywords: | Optic Atrophy, Hereditary, Leber Mitochondrial DNA -- Abnormalities Ophthalmic drugs industry |
| Issue Date: | 2018-11 |
| Publisher: | University of Malta. Department of Pharmacy |
| Citation: | Zuccarelli, M., Borg, J. J., Vella, J., & Serracino-Inglott, A. (2018, November). Developing safe and effective medicinal products to treat Leber hereditary optic neuropathy. Clinical and regulatory challenges. Poster session presented at the X Malta Medical School Conference, St Julian's, Malta. |
| Abstract: | Leber Hereditary Optic Neuropathy (LHON) is a rare maternallyinherited mitochondrial optic neuropathy caused by three mitochondrial DNA point mutations. Raxone (idebenone) is the only approved medicinal product to treat LHON (MPLHON) within Europe. In the United States (US), Raxone was granted an orphan designation1 and currently there are no FDA-authorised MPLHON. The aims of this study were: 1) To evaluate MPsLHON under development 2) To understand clinical and regulatory pathways pursued by pharmaceutical companies when developing MPsLHON |
| URI: | https://www.um.edu.mt/library/oar/handle/123456789/48525 |
| Appears in Collections: | Scholarly Works - FacM&SPha |
Files in This Item:
| File | Description | Size | Format | |
|---|---|---|---|---|
| Developing_safe_and_effective_medicinal_products_to_treat_Leber_Hereditary_Optic_Neuropathy_2018.pdf | 541.35 kB | Adobe PDF | View/Open |
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