In Mumbai, hematologist Dr Savita Rangarajan, who is attached to Somaiya Hospital and the University of Southampton, the UK, is working with patients’ groups to increase access to the newer modalities. The team is working on starting gene therapy clinical trials in Mumbai later this year.
Dr Rangarajan’s work with patients such as Kshitij has been published in ‘The Lancet’ as a part of a multicentre trial on fitusiran. “This is a revolutionary treatment for both haemophilia A and B, and the phase 3 results are very encouraging. The results showed significant decrease in the life-threatening or major bleeds in the majority of the patients,” said Dr Rangarajan.
“The advantage of these new therapies is excellent control of spontaneous and joint bleeds, decrease in hospital visits and admissions and hence improved quality of life,’’ said Dr Rangarajan.
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