Maria Elisa Mancuso: During Day 2 of the Annual EHC Conference We Discussed Very Rare Bleeding Disorders!
Maria Elisa Mancuso, Senior Hematology Consultant at Humanitas Research Hospital, shared on LinkedIn:
”During Day 2 of the Annual EHC Conference in Vienna we discussed very rare bleeding disorders as fibrinogen defects (thanks to Alessandro Casini) and the “newly recognized” group of Bleeding Disorders of Unknown Cause – BDUC (thanks to Johanna Gebhart).
Congratulations to Dominik Cepic for is wonderful overview and passionate advocacy for hemophilia B with inhibitors as “rare among rares”.
The SLAM session shed lights to many important issues as orthopedic evaluation and care of arthropathy, treatment of PUPs, pain and ITI.
During the session dedicated to gene therapy real world experience (Wolfgang Miesbach), struggles in procurement and reimbursement (Gaetan Duport) and new non-AAV mediated gene therapies (Paul Batty) were discussed.
Finally the day was beautifully close by the energy of the youth debates.”
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