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Genome sequencing of thousands of patients with rare blood disorders
Approximately 3M people have a rare bleeding disorder or disease of platelets, which are the cell fragments that help blood clot. The genetic causes of dozens of such disorders are known (e. g.
Read moreLong-lasting, late-onset, and chronic idiopathic neutropenia project, February 2025 update
A project update from Dr Grigorios Tsaknakis.
Read moreWithout access to treatment, can we truly innovate in rare diseases?
Interview with Giampaolo Merlini
There have been many developments on rare diseases since the EU Orphan Medicinal Products Regulation came into force in 2000. As the European Commission is evaluating its effectiveness, EHA discussed with Prof.
Collaboration with the EMA on Joint Clinical Assessments and Joint Scientific Consultations under the HTA Regulation
The European Hematology Association (EHA) welcomes the draft Implementing Act laying down procedural rules for the exchange of information between the Health Technology Assessment (HTA) Coordination Group, the European Commission, and the European Medicines Agency (EMA).
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