We just had a very interesting educational session on TA-TMA. I think it was very provocative as well and trying to move things into the future for this disease. We know that it’s a syndrome of high morbidity and mortality and it is an endothelial injury syndrome, the most well studied up to now. We are very fortunate that we have done some studies on the pathophysiology, focusing on the drivers of this syndrome...
We just had a very interesting educational session on TA-TMA. I think it was very provocative as well and trying to move things into the future for this disease. We know that it’s a syndrome of high morbidity and mortality and it is an endothelial injury syndrome, the most well studied up to now. We are very fortunate that we have done some studies on the pathophysiology, focusing on the drivers of this syndrome. It is a multifactorial syndrome, so we have complement activation, but also inflammatory markers there, endothelial damage as well. But all these studies have eventually moved the field forward to clinical trials of complement inhibitors that have shown safety and efficacy in a lot of patients. And I think this field is moving forward with new diagnostic criteria, but also response criteria. We are eagerly waiting for the results of all clinical trials as well to be published so that we know the results of all biomarkers how this can be adapted into clinical practice and all that. And in total it was very I think educational session and trying to show that we all work together to find the cure or the best path for our patients.
Dr Vasu provided a very nice talk for the real-world adaptation of the criteria and the adult population. We had also a talk from pediatric nephrologist Dr Hingorani trying to see their perspective and also a talk from Professor Rambaldi from the adult hematologist perspective. I think there were also very nice clinical case reports from patients that actually did benefit from narsoplimab as an FDA-approved complement inhibitor right now for this syndrome. I think in total we continuously try to learn about how we use this criteria in the real world and of course the criteria is not just something solid it can be transformed in the future to better fit our patients.
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