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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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Selected EMA news

February 2025New medicines recommended for approval
Dyrupeg (pegfilgrastim) – biosimilar
Treatment to shorten the duration of neutropenia and help prevent febrile neutropenia after cytotoxic chemotherapy

Eltrombopag Accord (eltrombopag) – generic
Treatment of primary immune thrombocytopenia and thrombocytopenia associated with chronic hepatitis C
New information on…

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