Haemophilia gene therapy patients thriving over a decade after treatment
Sanatate
Patients who received a one-off gene therapy to treat haemophilia are still doing well up to 13 years later, proving that the therapy is safe and long-lasting, finds a new study from researchers at UCL, the Royal Free Hospital and St Jude Children’s Research Hospital, Tennessee. The study, published in The New England Journal of Medicine , is the longest reported follow-up for any gene therapy for haemophilia B, a rare genetic disorder caused by insufficient levels of a protein in the blood,
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