The World Duchenne Organization is pleased to announce that the WDO Leadership Award, the highest recognition for individual achievement, was awarded to Professor Francesco Muntoni on September 7, 2022. Prof Muntoni is Director of the Dubowitz Neuromuscular Centre at Great Ormond Street Institute of Child Health and Great Ormond Street hospital. This award acknowledges and celebrates…
As a continuation of the funding of an EJP RD NSS call, ECRIN is holding a two-day workshop and networking event in Prague on October 4th & 5th; the event will see the gathering of investigators involved in drug repurposing academic-sponsored trials for rare diseases, as well as other rare diseases clinical trial stakeholders. To improve the existing treatments of…
IRDiRC, a global collaborative initiative with the vision to enable all people living with a rare disease to receive an accurate diagnosis, care, and available therapy within one year of coming to medical attention, today announced the formation of the Regulatory Science Committee (RSC). The new RSC “brings broad expertise across different stakeholders and geographies allowing…
EJP RD held its annual General Assembly and Consortium Meeting on 13-15 September 2022 in Porto, Portugal. The event gathered over 100 participants from various institutions across Europe and associated countries. It was an opportunity to bring back together EJP RD members to meet in-person. Collaboration, Innovation, Data, Funding & Empowerment are key for the…