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dc.contributor.authorKennedy, Cormac
dc.contributor.authorHennessy, Martina
dc.date.accessioned2024-01-29T14:27:28Z
dc.date.available2024-01-29T14:27:28Z
dc.date.issued2021
dc.date.submitted2021en
dc.identifier.citationKennedy C., O'Dea E., Reidy D., Towns J., Pigott M., Kevans D., O'Rourke D., Hennessy M., Challenging times: Delivering gene therapies and an opportunity for shared learning, British Journal of Clinical Pharmacology, 2021en
dc.identifier.issn13652125 03065251
dc.identifier.otherY
dc.identifier.urihttp://hdl.handle.net/2262/104814
dc.descriptionPUBLISHEDen
dc.description.abstractAs we embrace the era of precision medicine, gene therapies are the vanguard in the fight to cure rare diseases. While rare diseases are just that, rare, it is suggested that patients with rare diseases worldwide outnumber those with HIV and cancer combined.1 The development of safe and effective therapies that target genetically driven diseases has progressed significantly since the initial controversies. Now that gene therapies have moved from bench to bedside and achieved regulatory approval, a pipeline of therapies exists providing much needed hope for patients with incurable rare diseases. However, processes to customize the administration of these therapies in a safe, patient-centred and efficient manner are necessary for health care systems. Our recent experience delivering gene therapies, and Zolgensma® specifically, involved many challenges, not least an unprecedented COVID-19 pandemic. Following the recent themed issue on Advanced Therapies in this journal, our experience provides an opportunity to share lessons with international centres who will deliver these therapies.en
dc.language.isoenen
dc.relation.ispartofseriesBritish Journal of Clinical Pharmacology;
dc.rightsYen
dc.titleChallenging times: Delivering gene therapies and an opportunity for shared learningen
dc.typeJournal Articleen
dc.type.supercollectionscholarly_publicationsen
dc.type.supercollectionrefereed_publicationsen
dc.identifier.peoplefinderurlhttp://people.tcd.ie/mhenness
dc.identifier.peoplefinderurlhttp://people.tcd.ie/kennec30
dc.identifier.rssinternalid236037
dc.identifier.doihttp://dx.doi.org/10.1111/bcp.15125
dc.rights.ecaccessrightsopenAccess
dc.identifier.orcid_id0000-0002-2153-5288


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