Last week, Claudia and I from Factor-H, together with the Venezuelan physicians and patient association (Avehun), we spent a day visiting the neighborhood of San Luis, outside Maracaibo. We hand-delivered clothes donated by our US friends and colleagues, to about 50 people living with HD in their families. It was a day where at least we brought a smile and a bit of help to our Venezuelan friends living with HD. Factor-H also donated money to buy food. To all who helped Nacho gather the clothes, thank you very much, from Factor-H and from the patients!
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Factor-H
Uniqure’s AMT-130 Phase 1/2 trial – a reflection
Yesterday, Uniqure reported on the 24-month results of their Phase 1/2 clinical trial with AMT-130, a viral gene therapy trial