Tweeting for the first time today about ny gene therapy experience. I had FIX Gene Therapy vector in late february with Padua FIX gene in Phase 3 clinical trial 1/ https://www.irishtimes.com/news/health/irish-haemophilia-patients-given-functional-cure-1.4430168

The infusion day went very smoothly 2/

Follow up was weekly for the first 12 weeks and then monthly until year 1. Visits involved a significant blood draw each week. Despite the pandemic, the visits @stjamesdublin were very well organised and safe. Initially, visits took place in a seperate non clinical building 3/

My experience to date has been very positive. No adverse events, no increase in transaminases, no requirement for steroids. FIX expression up by week 1 and from week 11 to week 39 has been between 48% and 59%. No bleeding episodes and no factor use since the trial started 4/

Some 2 weeks ago, I dropped a heavy item on my bare foot. This would definitely have caused a bleed requiring treatment in the past. On thus occasion, iced the foot for 30 minutes. No bleed resulted. Quite a change after 62 years of living with severe haemophilia 5/

Joints feel good, ankle (fused) and knee (replaced) still give some discomfort and stiffness but this type of long term joint damage does not reverse. Nice not to have to remember and schedule regular IV injections.