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The European Medicines Agency (EMA) has recommended granting a conditional marketing authorisation in the European Union (EU) for Roctavian (valoctocogene roxaparvovec) for the treatment of severe haemophilia A in adults who do not have factor VIII inhibitors and no...
The European Medicines Agency (EMA) recommended granting a marketing authorisation in the European Union (EU) for Xenpozyme (olipudase alfa), Upstaza (eladocagene exuparvovec) and a marketing authorisation under exceptional circumstances for Zokinvy (lonafarnib). ...
We are pleased to launch our March newsletter. In 2022 we will continue to advance paediatric research initiatives. We have several exciting news: our podcast that will present all EPTRI’s activities and opportunities, our new website and Twitter account, new...
1) XVI European Patients’ Rights Day 2022. The "Conference on the Future of a Healthy Europe: Taking part in the decision-making process on health priorities” will be held on April 20th–21st 2022 from 14:30 to 16:00 CEST. The aim is to provide a multi-stakeholder...
EPTRI in collaboration with EJP RD is organizing this workshop on 4th-5th July 2022 in Bari, Italy. The course aimed at facilitating discussion and exchange of knowledge on the M&S methodologies as well as innovative/promising strategies for facing complex...
EPTRI would like to thank all speakers and partners for coming together to make this virtual event happen. Sessions are now available on-demand and can be watched via the following link that will give you access to the recordings of the live sessions. Please...
The Pharmaceutics journal dedicated this special issue to highlight this important event that was organized by EPTRI last December online. During this event 10 exciting presentations to sum up the activities carried out so far and the short and long-term ambitions and...
The 9th Congress of the European Academy of Paediatric Societies (EAPS) that will be held between October 7th-11th, 2022, in Barcelona, Spain. The Congress, which is organized by three European societies: the European Academy of Paediatrics (EAP), the European...
This meeting organized by the European Alliance for Transformative Therapies (TRANSFORM) MEP Interest Group, from which EPTRI is a member. It will be held online on Thursday, March 31st, 2022 from 15:00 to 17:00 (CET). Advanced Therapy Medicinal Products (ATMPs)...
In the context of EJP RD’s ERN Workshops, the “Translational research on bone impairment in rare diseases” workshop aimed at sharing updates on this important topic by bringing together experts and trainees to facilitate collaborations. It is being organized...