News

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 consultation on the draft document from September 2019 until November 2019, with the aim of identifying potential gaps and

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 medicines in this area. Now, the revision of the Regulations will be open for

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 regarding their health”. It constitutes an important step in drawing the pathway

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 consultation on the draft document from September 2019 until November 2019, with the aim of identifying potential gaps and

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 the ‘late infantile’ or ‘early juvenile’ forms of MLD, who have been identified as carriers of

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 diseases. Existing tools such as orphan designation, protocol assistance and PRIME scheme will be highlighted and

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 global advocacy of the community. Due to pandemic, this year the event will be

  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 aspects of clinical management and health care, for this reason Harvard Medical School (HMS) is providing

An early announcement of the joint transnational call 2021 has been published on EJP RD website. In order to improve healthcare system and ordinary life of people that live with rare diseases, this call has the aim to encourage research on Social Sciences and Humanities (SSH). Of high relevance during the call, as it can

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 goal of this project, as stated in the guide, is to encourage a prolific and