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Without 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.
COST Action ‘EuNet-INNOCHRON’
The European Cooperation in Science and Technology (COST) is an EU-funded, intergovernmental framework that aims to create pan-European research networks in all science fields and promote excellence, foster interdisciplinary research and empower young researchers and innovators.
Read moreAddressing the bureaucracy challenge
EHA has recently brought key stakeholders around the table to discuss bureaucratic obstacles in clinical research.
Read moreNew data exchange platform on rare diseases
The European Commission (EC) launched a new online knowledge-sharing platform – the European Platform on Rare Disease Registration (EU RD Platform) – on February 28, 2019.
Read moreMission ‘Gene Therapy’ for Horizon Europe
Mission ‘Gene Therapy’ for Horizon Europe
Advanced therapies, such as gene therapy, hold promise for treating a wide range of chronic diseases and improving patients’ quality of life.
Uptake of CAR T cell therapy in Europe
Gene (editing) therapy continues to break ground in hematology, showing promising results in tackling severe blood cancers. Yet, its uptake in the European Union is slow, with few patients benefiting from it so far.
Read moreHospital pharmacists and regulators lead the way on shortages
EHA welcomes initiatives but calls for more focus on causes
Medicine shortages have a negative impact on the quality and cost of treatments and on patient access to the best possible care.
Major changes needed for patients to benefit from precision medicine
Precision medicine approaches the treatment of a disease via an individual’s genes, environment, and lifestyle.
Read moreEHA-funded study in The Lancet Haematology: Economic Burden of Blood Disorders in EU is €23 billion
In Europe blood disorders affect around 80 million people. The total cost of blood disorders consists of healthcare expenditure (€15. 6 billion), productivity loss due to illness and mortality (€5. 6 billion), and the costs of informal care (€1.
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