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Please use this identifier to cite or link to this item: https://www.um.edu.mt/library/oar/handle/123456789/48525
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dc.date.accessioned2019-11-08T10:29:38Z-
dc.date.available2019-11-08T10:29:38Z-
dc.date.issued2018-11-
dc.identifier.citationZuccarelli, 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.en_GB
dc.identifier.urihttps://www.um.edu.mt/library/oar/handle/123456789/48525-
dc.description.abstractLeber 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 MPsLHONen_GB
dc.language.isoenen_GB
dc.publisherUniversity of Malta. Department of Pharmacyen_GB
dc.rightsinfo:eu-repo/semantics/openAccessen_GB
dc.subjectOptic Atrophy, Hereditary, Leberen_GB
dc.subjectMitochondrial DNA -- Abnormalitiesen_GB
dc.subjectOphthalmic drugs industryen_GB
dc.titleDeveloping safe and effective medicinal products to treat Leber hereditary optic neuropathy. Clinical and regulatory challengesen_GB
dc.typepresentationen_GB
dc.rights.holderThe copyright of this work belongs to the author(s)/publisher. The rights of this work are as defined by the appropriate Copyright Legislation or as modified by any successive legislation. Users may access this work and can make use of the information contained in accordance with the Copyright Legislation provided that the author must be properly acknowledged. Further distribution or reproduction in any format is prohibited without the prior permission of the copyright holder.en_GB
dc.description.reviewedN/Aen_GB
dc.contributor.creatorZuccarelli, Marta-
dc.contributor.creatorBorg, John-Joseph-
dc.contributor.creatorSerracino-Inglott, Anthony-
dc.contributor.creatorVella, Janis-
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