Hemophilia gene therapy—New country initiatives
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DOI: 10.1111/hae.14080
Abstract: Gene therapy is an opportunity for haemophilia patients to receive a one‐time treatment and have lasting factor levels for years or decades instead of dependence on repeated administration within short intervals and on sustained supply… read more here.
Keywords: haemophilia; gene therapy; hemophilia gene; therapy new ... See more keywords
The Hemophilia Gene Therapy Patient Journey: Questions and Answers for Shared Decision-Making
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DOI: 10.2147/ppa.s355627
Abstract: Purpose The anticipated emergence of hemophilia gene therapy will present people with hemophilia (PWH) and treating clinicians with increasingly complex treatment options. It will be critical that PWH and their families be empowered to participate… read more here.
Keywords: gene therapy; hemophilia gene; journey; decision ... See more keywords
Hemophilia Gene Therapy: From Bench to Bed Side.
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DOI: 10.2174/1566523219666190924121836
Abstract: Novel gene therapy strategies have changed the prognosis of many inherited diseases in recent years. New development in genetic tools and study models has brought us closer to a complete cure for hemophilia. This review… read more here.
Keywords: gene therapy; therapy bench; hemophilia gene; bench bed ... See more keywords