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The elimination of the need for continued prophylaxis, or factor replacement following trauma or prior to surgery would lead to annual cost savings. Such optimized health and well\u2010being would be reaching a level of health equity that was unimaginable several decades ago. \u2018Before anything else, preparation is the key to success\u2019\u2014Alexander Graham Bell. This quote from the famous inventor, scientist and engineer highlights that, although we currently stand on the threshold of this achievement, delivering on this promise will require broad\u2010based multistakeholder preparation (scientists, manufacturers, federal regulators, health technology assessors, persons with haemophilia, national advocacy groups and multidisciplinary healthcare teams) with a focused emphasis on <jats:italic>education<\/jats:italic>, approval of <jats:italic>safe and effective<\/jats:italic> therapies, removal of barriers to <jats:italic>access<\/jats:italic> and excellence in <jats:italic>clinical delivery<\/jats:italic>.<\/jats:p>","DOI":"10.1111\/hae.14027","type":"journal-article","created":{"date-parts":[[2020,6,3]],"date-time":"2020-06-03T09:02:35Z","timestamp":1591174955000},"page":"114-121","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":24,"title":["Delivering on the promise of gene therapy 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