The TEDDY Chair, Annagrazia Altavilla, consultant and expert of the Council of Europe, is very pleased to announce the approval by the Committee on Bioethics of Council of Europe of a Concept note on “Participation of children in the decision-making process on matters...
On 25 November 2020, the European Commission published the inception impact assessment on the revision of the Orphan and Paediatric Regulation. EU legislation on medicines for children and rare diseases was introduced in the early 2000’s, to address the lack of...
On 31 August 2020, the Enpr-EMA (European Network of Paediatric Research at the European Medicines Agency) working group on trial preparedness published recommendations on preparedness of medicines’ clinical trials in paediatrics. Enpr-EMA held a two-month public...
The first General Assembly of the EPTRI (European Paediatric Translational Research Infrastructure) Italian Node (EPTRI-IT) was held online on October 28th, 2020. The 3 hours meeting was attended by more than 60 representatives coming from the 24 Italian Institutions...
On 30 November 2020, the European Medicines Agency will organise a workshop on support for orphan medicines development with the aim to encourage early and efficient interactions with the regulators by highlighting pre-marketing support in medicine development in rare...
EMA has recommended granting a marketing authorisation in the European Union for the gene therapy Libmeldy to treat metachromatic leukodystrophy (MLD), a rare inherited metabolic disease that affects the nervous system. Libmeldy is indicated for use in children with...
On 31 August 2020, the Enpr-EMA (European Network of Paediatric Research at the European Medicines Agency) working group on trial preparedness published recommendations on preparedness of medicines’ clinical trials in paediatrics. Enpr-EMA held a two-month public...
On November 30 until December 6, 2020, the Duchenne Data Foundation has organised its third Edition of the “Duchenne Patient Academy”. The Duchenne Patient Academy has the aim to create a wider understanding of Duchenne Muscular Dystrophy (DMD) and coordinate the...
The Harvard Medical School is releasing a series of educational videos covering the COVID-19 pandemic. This initiative started on April 2020 and was designed especially for physicians, nurses and other health care providers. COVID-19 continues to change many...
The European Joint Programme on Rare Diseases (EJP RD) has developed, together with a working group led by EURORDIS, named Patient Engagement in Biomedical Research Projects (PENREP), a short guide on Patient Partnership in rare disease research projects. The main...