The U.S. Food and Drug Administration (FDA) has recently published a guidance on neonatal clinical pharmacology studies. This guidance is intended to assist sponsors of investigational new drug applications (INDs) as well as applicants of new drug applications...
News
Current State of Paediatric Reference Intervals and the Importance of Correctly Describing the Biochemistry of Child Development: A Review
Good paediatric biomarkers are critical for the decision-making process in child healthcare. However, reference intervals used in pediatric care today remain highly inconsistent across a broad range of common clinical biomarkers. This review paper assessed...
EMA has recommended granting a conditional marketing authorization to the first gene therapy to treat Haemophilia A
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...
EMA has recommended granting marketing authorisation to the first three therapies for Rare Disorders in the European Union
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). ...
New EPTRI newsletter is out!
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...
The Active Citizenship Network has organized some interesting initiatives focused on health in April and May 2022
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...
Modeling & Simulation (M&S): Research Methodologies for Small Populations in Rare Diseases
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...
1st International Conference on Rare Diseases and Paediatric Research
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...
Special Issue “Scientific Highlights in the First European Paediatric Translational Research Infrastructure (EPTRI) Open Meeting”
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...
9th Congress of the EAPS: “Shaping the Future of Child Health”
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...