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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.
Publications
Dexamethasone treatment for COVID-19 is related to increased mortality in hematologic malignancy patients: results from the EPICOVIDEHA registry
Aiello TF, Salmanton-Garcia J, Marchesi F, et al. Haematologica. 2024 Aug 1;109(8):2693-2700.
Other COVID-19 Resources
Safety of COVID-19 vaccines – April updates
ECDC and EMA issue advice on fourth doses of mRNA COVID-19 vaccines
EMA recommends authorisation of COVID-19 medicine Evusheld
EMA recommends authorisation of booster doses of Comirnaty from 12 years of age
EMA recommends approval of Spikevax…
EU calls for proposal 2023
IntroductionHorizon Europe is the research and innovation programme of the EU for the period 2021-2027. The programme facilitates research collaboration and strengthens the impact of R&I in developing, supporting and implementing EU policies while tackling global challenges.
Read moreUpdate on EU4Health Calls and Contracts for 2024
The European Health and Digital Executive Agency (HaDEA) has published the tentative calendar for the EU4Health program calls and contracts for 2024.
Read moreSWG Educational Activities
EICMLThe 2023 EICML meeting took place in Naples in May 2023. Fifty-five physicians and scientists met at the Palazzo Alabardieri in central Naples to spend two days discussing emerging data and developing collaborations.
Read moreSelected 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…
Mini-hormone fights iron
Iron overload commonly leads to organ damage and even death. Currently available treatments for iron overload are burdensome or cause side effects.
Read moreFirst randomized evidence for kinase inhibitor activity in acute myeloid leukemia
Despite the success of tyrosine kinase inhibitors in some forms of leukemias such as chronic myeloid leukemia and acute lymphoblastic leukemia, until now a kinase inhibitor had yet to demonstrate activity in acute myeloid leukemia (AML).
Read moreAcute Lymphocytic Leukemia: impressive results with the monoclonal antibody blinatumomab
The Phase 2 dose-ranging study MT103-206 evaluated the efficacy, safety and tolerability of blinatumomab in adult patients with B-precursor Acute Lymphoblastic Leukemia who had relapsed following treatment with standard front-line chemotherapy or allogeneic stem cell transplant.
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