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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.

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Position of EHA on Research Funding

 

The challenge
The cost of biomedical research is great, but the cost of disease is immense. An ageing population and expensive innovations in medicine put an increasing burden on already stressed healthcare budgets.

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How malignant cells in patients with Chronic Lymphocytic Leukemia escape T cell recognition and attack

T cell activation is essential for immunity including the recognition and killing of abnormal target cells such as cancerous cells.

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Writing and Publications WG

Responsibilities:
Authoring project and consensus papers.

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Data Analysis and Interpretation WG

Responsibilities:
Managing and analyzing data centrally. Planning and implementing the necessary infrastructure and data transfer agreements.

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Preanalytical WG

Responsibilities:
Reviewing SOPs for sample handling and storage to ensure uniformity across the consortium. Overseeing the logistics of sample collection. Defining the minimum (core markers) and extended panels for immunophenotyping. Establishing good practice sampling for retrospective analysis.

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Clinical WG

Responsibilities:
Identifying the clinical data required for collection. Locating clinical trials where suitable samples will be collected or have already been collected. Involvement in the European Registry. Assessing the type of database and dataset currently available.

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