The evolving landscape of gene therapy for congenital haemophilia: An unprecedented, problematic but promising opportunity for worldwide clinical studies.
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DOI: 10.1016/j.blre.2020.100737
Abstract: With liver-directed gene therapy, congenital haemophilia has the potential to progress from an incurable to a phenotypically curable condition. However, the proportion of haemophilia population likely to benefit from gene therapy remains to be established.… read more here.
Keywords: gene; gene therapy; congenital haemophilia; therapy congenital ... See more keywords
Evolution of congenital haemophilia care in Taiwan.
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DOI: 10.1016/j.jfma.2021.07.017
Abstract: Haemophilia care in Taiwan has come a long way over the past 35 years, from the absence of specialised haemophilia treatment centres before 1984 to the establishment of treatment centers in the majority of medical… read more here.
Keywords: care taiwan; evolution congenital; congenital haemophilia; haemophilia care ... See more keywords
Congenital haemophilia A presenting with subgaleal and intracranial haemorrhage following instrumental delivery
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DOI: 10.1136/bcr-2021-248030
Abstract: © BMJ Publishing Group Limited 2022. No commercial reuse. See rights and permissions. Published by BMJ. DESCRIPTION The case is of a male infant born by vacuumassisted vaginal delivery at 38 weeks+3 days of gestation.… read more here.
Keywords: time; haematoma; age hours; congenital haemophilia ... See more keywords